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Foundation NewsLine and Recent Website Updates


Archived Newsline Postings from 2014, 2013 & 2012




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This week, I would like to highlight a "special" couple who took the opportunity to raise awareness into Niemann-Pick Disease on their wedding day on behalf a precious loved one in their family.  Please read below to learn more about this "self-less" pay-it-forward....act of wedding day kindness!

A quick reminder to all of our family members, their extended family and community supporters ~ don’t forget to let us know about your fundraising or awareness events as soon as you have targeted your event and the date! 

The NNPDF Central Office is here to help you in all aspects of your campaigns.  Either contact the NNPDF Central Office and ask to speak with Chris for information on what the NNPDF can do for you ~ or ~ check out the Fundraising and NPD Awareness Services Plan at http://www.nnpdf.org/documents/FundraisingServicesPlan.pdf .

Need any assistance? Simply contact Chris Klauer, Family Services & Marketing Coordinator at nnpdf@nnpdf.org

http://tollberghomes.net/wp/wp-content/uploads/2015/06/congratulations_011.jpg

 

The National Niemann-Pick Disease Foundation would like to congratulate and thank Nici & Craig Peterson on their recent wedding ceremony!  Nici & Craig asked that in lieu of wedding gifts, their loved ones and guests direct donations to the NNPDF, in loving honor and support of Garrett Hopkin (NPB) and the work of the foundation.

Garrett, is Nici's sweet nephew. Pictured below is Garrett with his beautiful Aunt Nici, as well as, the loving couple!  It certainly appears as though all had a great time at the wedding!  The NNPDF is so thankful and appreciative for this couples kind consideration of the foundation and the work we support.  Heartfelt thanks ~ go out to Nici & Craig!!!

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Aunt Nici and Garrett Nici & Craig

 

[Jan 18th, 2016 ~ blg]

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Jan1516

From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator

Here in Wisconsin we just finished with a cold snap and warmed up to 35 degrees today.  However another cold snap is just around the corner.  One of my favorite photographers took the picture of the ice caves near Apostle Islands National Lakeshore.  Thought you might like to see some of the beauty of Wisconsin winters.

Island
© Copyright 2015, Life in the Northwoods

My "Family Support Services" update this week highlights:

  • 24th Annual NNPDF Family Support and Medical Conference
  • Now I Lay Me Down To Sleep
  • Infinity Coaches’ Charity Challenge to raise money for NPC research
  • CTD Press Release

Please feel free to contact me at: nnpdf@nnpdf.org or give me a call at: 1-877-287-3672 if you have any questions pertaining to the work and “Family Support Services” afforded to the members of the foundation.  If you find a great resource, please pass it along so I can share it with the rest of our NNPDF Family Membership!

Have a great weekend!

Click here to view the full post

Chris Klauer
NNPDF Family Support and Marketing Coordinator

[Jan 15th, 2016 ~ blg]

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Fin

Dear NNPDF Families and Friends,

As we wrap-up year-end 2015 and embark on all that 2016 has in store for the NNPDF, the Board of Directors wanted to provide you with a Finance Committee update.  The Finance Committee is responsible for continually reviewing the financial affairs of the NNPDF during the year, preparing the budget, reviewing any anticipated expenses, and communicating the financial condition of the NNPDF to the Executive Committee and NNPDF Board of Directors. 

Please find a year-end 2015 update from the NNPDF Finance Committee Chair, Jill Flinton, of Charlton, New York.  Jill serves on the foundation board in a volunteer capacity and in memory of her son, Daniel (NPC) who passed away from on November 11, 2011 at the tender age of four years.

To access the NNPDF Foundation Financials and Forms web page directly ~ simply go to the NNPDF Web Site Home Page at www.nnpdf.org, choose About Us, follow the link to the NNPDF Financial Files and Form to see most recent update post to the NNPDF membership; dated: Jan 12th, 2016 or follow this direct link: http://nnpdf.org/aboutus_02.html#FinCommittee2

Please find “quick links” to all of these previous posts below.  

If you should have any questions related to this information and/or update ~ please feel free to contact the foundation for assistance.

[01/12/2016 ~ blg]

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Save the Date

The NNPDF is excited to announce that the 24th Annual Family Support & Medical Conference will be held in Boston, Massachusetts on August 11-14, 2016.

Specific details associated with this summer 2016 event are being finalized and will be posted as soon as they become available!

For now, please pencil in these dates on your summer calendar ~ and ~ we hope to see you all there!

[01/11/2016 ~ blg]

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$100,000 for NPC Research Infiniti Coaches Charity Challenge ~ 2016
January 4th ~ March 29th, 2016

Dear NNPDF Families and Friends,

The entire Niemann-Pick Disease type C  community has been offered an unparalleled opportunity to raise $100,000 towards NPC research, but it will only happen if YOU take action and encourage others to do the same. It's easy! Vote once a day online between now and March 29th to push the Trent and Julie Smith Family Foundation "BReaK Thru Fund" for NPC research to the finish line and the top prize of $100,000! (Three of the Smith family children have been diagnosed with Niemann-Pick Disease type C ~ Braden, Riley and Keaton ~ thus BReaK-Thru Fund.)

Painter

Click Here to Vote for Matt Painter!

The first round of voting opened on January 4th, 2016 and continues through January 29th, 2016 when 24 Coaches out of the initial 48 will advance to round two of four total.  Vote daily for Matt Painter to ensure he makes it to the final round!

Share this with your families, share this with your friends and encourage them to do the same.
Alone we are rare, but together we are strong!  


www.votemattpainter.com

[01/11/2016 ~ blg]

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From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator

My update this week highlights:

    • Family Voices
    • Understanding Breakthrough Therapy Designations
    • NPD “In the news!”

Please feel free to contact me at: nnpdf@nnpdf.org or give me a call at: 1-877-287-3672 if you have any questions pertaining to the work of the foundation.  If you find a great resource, please pass it along so I can share it with the rest of our NNPDF Family Membership!

Click here to view the full post: http://www.nnpdf.org/FamilyServiceSupport.html#Jan082016

Have a great weekend!
Chris Klauer
NNPDF Family Support and Marketing Coordinator

[Jan 8th, 2016 ~ blg]

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Vtesse, Inc. Announces FDA’s Granting of Breakthrough Therapy Designation for
VTS-270
(2-hydroxypropyl-β-cyclodextrin) in Niemann-Pick Type C1 Disease

Dateline: 01/06/2016

Vtesse

The National Niemann-Pick Disease Foundation (NNPDF) is pleased to share with our NPC Community Members that the Food and Drug Administration (FDA) has granted VTS-270 (2-hydroxypropyl-β-cyclodextrin) a Breakthrough Therapy Designation. The FDA Breakthrough Therapy designation is designed to expedite the development and review of drugs within the FDA regulatory process.

This designation is granted by the FDA when the preliminary clinical data indicates that the drug may demonstrate substantial improvement on clinically significant endpoint(s). The Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review, and will allow for a close collaboration between Vtesse and the FDA on the VTS-270 (2-hydroxypropyl-β-cyclodextrin) development program.

Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial. For additional information regarding VTS-270 (2-hydroxypropyl-β-cyclodextrin) clinical trial, visit Vtesse's website.

Click here to Read the Press Release dated Jan 6th, 2015


Recording & Slides of Dec. 14th Webinar Now Available

In addition to the above press release, the NNPDF Central Office recently obtained the Webinar recording and slides associated with the Vtesse “Town Hall” Webinar hosted for the NPC patient community on December 14th, 2015.

Click here for the Webinar Recording & Slides

[Jan 6th, 2016 ~ blg]

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We Will Persevere!

NY

As 2015 draws to a close, families and friends will gather to celebrate all of the milestones afforded them throughout 2015.  As we all take the time to reflect and close the last chapter of 2015, we all look ahead to the new chapters which await us in 2016 ~ there are new advances and milestones awaiting our patients and families! 

Often, for our NPD families, the future is guided by the fear and trepidation of what awaits as the progression of Niemann-Pick Disease inevitably begins to take its toll on their loved ones.  BUT ~ as we look ahead towards 2016 ~ it is with a glimmer of hope that the ongoing and upcoming clinical trials for a number of therapies into NPD will be successful in slowing the disease progression.

We ask that the members of the foundation draw strength from the prose of Helen Keller, Your success and happiness lies in you. Resolve to keep happy, and your joy and you shall form an invincible host against difficulties.

To cap off 2015, we have created a “Year-end” e-Newsletter to share with the NNPDF community. 

 It includes:

  • Thanks for your ongoing financial support in 2015 ~ “Year-end” donation reminders!
  • In Memoriam ~ Remembering Those We Lost in 2015
  • Coping with Loss During the Holidays
  • 2015 E-Newsletters Recap
  • 2016 Annual NNPDF Board Meeting

Click here to view the 2015 End of Year e-Newsletter!

As the New Year approaches with hopes anew, the NNPDF wishes to extend to you and your family a wonderful year ahead.  Happy New Year!

[Dec 31st, 2015 ~ blg]

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Giving the Gift of Hope in 2016!

During these tight fiscal times, we understand that there are many worthy causes vying for your donation dollars.  You can be assured that your gift to the NNPDF will be greatly appreciated and faithfully stewarded to bring the greatest benefit to families dealing with Niemann-Pick Disease in 2016 and beyond!

https://scontent-ord1-1.xx.fbcdn.net/hphotos-xpa1/t31.0-8/10380462_1496564243982447_6080523289224197937_o.jpg
Johnathan Spencer, age 9 ~ NPC ~
National Institutes of Health Phase 1 Clinical Trial Participant

Thank you in advance for your help towards our Quest for a Cure and supporting the National Niemann-Pick Disease Foundation (NNPDF) so that we can provide the gift of hope for our NPD patients and their families.

To donate now, simply follow the link below:

Donate Now button


There is, like never before, hope for the future ~ a future where Niemann-Pick Disease is no longer a threat to a full and productive life for patients and their families.

The NNPDF’s accomplishments of the past 23 years are due to the efforts of parents and families desperate to save their children from the devastating that is Niemann-Pick.


Giving the Gift of Hope ~ Legacy of Hope & Donations

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With the end of the year right around the corner, it reminds us of how far we've come in 2015 thanks to the ongoing work and research generously funded by our Niemann-Pick Friends and Families.

There are currently several promising NPD clinical trials in progress, which could lead to live-saving therapies. The NNPDF continues to fund promising research laboratories and we continue to raise awareness into NPD across the country. All of these efforts combined help us to continue moving towards that"Legacy of Hope" for a brighter future and eventually, a cure for all.

Here is how you can help give the gift of Hope for 2016!

Every little bit helps, even if you can only volunteer your time.  We understand not everyone can give money, which is why your support in any capacity is every bit as important.

Your contributions will not only assist us as we work to provide support to our families, but also enable us to offer the gift of hope through research!

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Don't forget to make your tax deductible donation
before December 31st, 2015!

All donations postmarked by December 31st, 2015 will receive a tax deductible receipt for 2015!

Donate Now!

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Thanks for your continued financial support in the fight against Niemann-Pick Disease!
Please contact us if you have any questions or need further information.

The NNPDF is a non-profit 501( c) (3) foundation and your gift is fully tax-deductible to the limit of IRS rules and regulations.
Tax ID #35-1844264 // www.nnpdf.org

[Dec 30th, 2015 ~ blg]

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The National Niemann-Pick Disease Foundation would like to wish you all season's greetings! 

Whatever is beautiful, Whatever is meaningful, Whatever brings you happiness...May it be yours this Holiday Season.

Best wishes to all of our Niemann-Pick Disease Foundation members and their supporters. 

May the warmth of hearth and home fill your hearts with joy this holiday season! 

~ The NNPDF Office Staff and Members of the Board of Directors

[Dec 22nd, 2015 ~ blg]

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NPC Vtesse Clinical Trial & Central Office Updates

Dateline: Dec 21st, 2015

 

Dear NNPDF Family & Friends,

The National Niemann-Pick Disease Foundation (NNPDF) is pleased to be able to share with our NPD community families updates associated with the following:

  • Vtesse VTS-270 NPD Type C Clinical Trial UPDATES
  • NNPDF Equipment Exchange Program and NEW posted items

 

Click here to view the NNPDF e-Blast

[Dec 21st, 2015 ~ blg]

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From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator

Dear NNPDF Families and Friends,

The Holidays are coming!  The Holidays are coming!  No don’t run for the hills!  Instead check out the Holiday Survival Guide that I found!

My update this week highlights:

  • Holiday Survival Guide
  • NNPDF & CCNNPDF Membership Update

Please feel free to contact me at: nnpdf@nnpdf.org or give me a call at: 1-877-287-3672 if you have any questions pertaining to the work of the foundation.  If you find a great resource, please pass it along so I can share it with the rest of our NNPDF Family Membership!

Have a great weekend!

Click here to view this week's Family Services Update post

Chris Klauer
NNPDF Family Support and Marketing Coordinator

[Dec 18th, 2015 ~ blg]

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Ghazi Tamim (NPA)
10/21/2013 - 12/07/2015

Ghazi

Dear NNPDF Families and Friends,

The NNPDF Central Office today received notice of the passing of Ghazi Taimur (10/21/2013 – 12/7/2015 ~ NPA). 

Ghazi’s father Taimur writes:  “We are saddened to inform all that our beloved son Ghazi, after 2 ½ years of fighting NPA died on 7th of December 2015.  Please remember him in your prayers.

To Ghazi’s loving parents, Taimur and Fatima please know that your NNPDF family is here for you and to let us know if you need anything in the coming days, weeks and months ahead. We are holding you all in our hearts and thoughts.

[Dec 16th, 2015 ~ blg]

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Year-end Shopping for the Gift of Hope!

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Dear NNPDF Families and Friends,

As year-end approaches and shopping for loved ones begins to hit “fever-pitch” level….more and more of us are turning to on-line shopping services for assistance in finalizing our Santa wish list!  If you have found yourself letting your “fingers do the shopping on-line” ~ we have an easy way in which your shopping efforts can benefit the NNPDF and our programs at the same time.

The NNPDF has taken steps this holiday season and throughout the entire year, to provide the opportunity for our supporters who enjoy on-lining shopping the ability to “Gift of Hope for a Brighter Future” for all those diagnosed with Niemann-Pick Disease (NPD). 

Click here to view the NNPDF e-Newsletter highlighting ways you can shop to support the Foundation

[Dec 15th, 2015 ~ blg]

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Cassandra Samborski (NPB)
01/07/1994 ~ 12/9/2015

Cassie

Dear NNPDF Families and Friends,

It is with a heavy heart that we notify our NPD family membership of the passing of Cassandra “Cassie” Samborski (January 7, 1994 – December 9, 2015 ~ NPB), who courageously fought the NPB battle for 21 years. Cassie loved traveling and spending time with her family.  She enjoyed making others happy and especially enjoyed connecting with other NPB patients and their families.  Cassie always had a bright smile and loving welcoming embrace to family and friends alike.

Cassie’s Dad, Wayne Samborski, shared the following update to the NPD community; For those of you that did not know Cassie, she was a pioneer of Niemann-Pick type B. Her disease back when first diagnosed was moving very fast and doctors gave her maybe a few years to live. At that time, she was the first person in the USA to undergo a Bone Marrow Transplant for Niemann-Pick type B. After two tries it finally worked. After a test showed she had the missing enzyme and was cured from Niemann-Pick type B. Her heart, liver, lungs and kidney paid a price and did get damaged but she made it 21 years instead of 5 years.”

To Cassie’s loving parents, Wayne and Candy, and beloved older sisters, Nicole & Jessica, please know that your NNPDF family is here for you and to let us know if you need anything in the coming days, weeks and months ahead. We are holding you all in our hearts and thoughts.

A link to Cassie’s obituary and details for the visitation and funeral are located at: Here

[Dec 11, 2015 ~ blg]

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ALERT: New Recruiting Sites for VTS-270 (2-hydroxypropyl-β-cyclodextrin) for NPC
Dateline: 12/09/2015

Vtesse

ALERT: United States NPD Type C Families & Friends

The NNPDF central office noted today that additional investigational sites for Vtesse’s VTS-270 (2-hydroxypropyl-β-cyclodextrin) Phase 2b/3 clinical trial for NPC has been updated and is now actively recruiting patients at the approved clinical trial centers in Orange, CA & Allentown, PA.

Families should follow enrollment criteria as provided on the www.clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit www.clinicaltrials.gov and refer to study reference number: NCT02534844

For all previous updates regarding this clinical trial, visit the the links below:

Vtesse Phase 2b/3 Clinical Trial Historical Timeline can be viewed: http://www.nnpdf.ca/Vtesse.html
NIH Phase 1 Clinical Trial Historical Timeline can be viewed: http://www.nnpdf.ca/Cyclodextrin.html

[Dec 9th, 2015 ~ blg]

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Kaitlyn Bexton (NPC)
01/24/2001 ~ 12/8/2015

Kaitlyn

Dear NNPDF Families and Friends,

It is with a heavy heart that we notify our NPD family membership of the passing of Kaitlyn Bexton (January 24, 2001 – December 8, 2015 ~ NPC), who courageously fought the NPC battle for 14 years. Kaitlyn loved watching “Dancing with the Stars”, listening to music and spending time with her family.  Katie had an infectious smile and filled a room with her spirited, happy and playful energy. 

Kaitlyn’s family takes comfort in the fact that she is now playing with her sister Ally (Alexandra), who also suffered from Niemann-Pick Disease Type C and passed away in 2009 at the age of 10. 

To Kaitlyn’s loving parents, Stasha and Michael, and beloved younger sister, Samantha, please know that your NNPDF family is here for you and to let us know if you need anything in the coming days, weeks and months ahead. We are holding you all in our hearts and thoughts.

To view Kaitlyn's service information and send condolences to the family: Click Here

[Dec 8th, 2015 ~ blg | Updated: Dec 10th, 2015]

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RE:  Register for Webinar on VTS-270 (2-hydroxypropyl-β-cyclodextrin) for NPC
Dateline: 12/07/2015

Vtesse


Register:  US and Canadian Audience, 12:30 PM EST*, December 14, 2015
https://attendee.gotowebinar.com/register/7538191617966914050

*9:30am Pacific, 10:30am Mountain, 11:30am Central, 12:30pm Eastern

Dear NNPDF Niemann-Pick Type C (NPC) Family Members,

The National Niemann-Pick Disease Foundation (NNPDF) is pleased to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse, Inc. with regards to the VTS-270 (2-hydroxypropyl-β-cyclodextrin) clinical trial for Niemann-Pick Disease, Type C (NPC). 

Please join Vtesse on December 14, 2015 where Dr. Paul Gissen and Dr. Elizabeth Berry-Kravis will be providing an update on the clinical trial and will be available to address your questions. Vtesse is committed to keeping the community fully informed and soliciting feedback as they move through the clinical trial process. 

Visit the Vtesse Clinical Trial page to read the full post and for more information on registering for the webinar: http://www.nnpdf.org/Vtesse.html#Dec072015

Sincerely,

Nadine M. Hill

Executive Director
National Niemann-Pick Disease Foundation

Vtesse Phase 2b/3 Clinical Trial Historical Timeline can be viewed: http://www.nnpdf.org/Vtesse.html
NIH Phase 1 Clinical Trial Historical Timeline can be viewed: http://www.nnpdf.org/Cyclodextrin.html

[Dec 7th, 2015 ~ blg]

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Dear NNPDF Families and Friends,

New for 2015, the NNPDF Board of Directors rolled out a new communication strategy which we hope will assist you in feeling more connected with the work of the foundation and your Board Representatives. This new communication strategy offers the foundation a unique opportunity to share on-going efforts, as well as new and existing programs, projects and activities being addressed on your behalf, our family membership and NNPDF key stakeholder’s.  Crucial to the role of the foundation is that of “communication liaison” between pharmaceutical and research agencies working to bring innovative therapies to our NPD patient community via clinical trials.
 
Today, we wish to share with you an update from the 2015 Research Committee Chair, Elissa Miller-Visoky, of Marlboro, New Jersey.  Elissa serves on the foundation board in a volunteer capacity and in honor of her son, Jack Visoky (NPB).

To access the NEW web page titled:  “Message from the NNPDF Board” ~ simply go to the NNPDF Web Site Home Page at www.nnpdf.org for the most recent update to the NNPDF membership; dated: Dec 4th, 2015 and or follow this direct link: http://www.nnpdf.org/aboutus_02.html#ResComm

2015 NNPDF Board Communication Recaps:

  • The NNPDF Board of Directors (BoD’s) wrapped up our annual board meeting in early March 2015 in Chicago, Illinois. 
  • On Tuesday, March 24th , 2015, the first update was penned by the sitting 2015 NNPDF Board Chair, Leslie Hughes, which was followed by the Nominations Board Chair, Lisa Chavez's post on March 31st, 2015. 
  • On April 16th, 2015 the foundation Finance Chair, Jill Flinton, shared her background and updates on the financial condition and efforts of the NNPDF, while the Development Committee Chair, Becky McGuire, told of all the exciting fundraising and development plans for 2015. 

Please find “quick links” to all of these previous posts below.

If you should have any questions related to this information and/or update ~ please feel free to contact the foundation for assistance.

[Dec 4th, 2015 ~ blg]

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NPD Type B ~ Clinical Trial Updates

genzyme

Dear Niemann-Pick Disease Type B Families ~

One of the key services of the National Niemann-Pick Disease Foundation (NNPDF) is to ensure that our patient community is provided with all of the available critical information needed to make fully informed decisions about moving ahead with medical treatment and/or participation in a clinical trial. 

Currently, there are two clinical trials being supported by Genzyme, a Sanofi Company, for our NPD Type B (ASMD) patient community.  Listed below are links to the available informational and resources web pages detailing specifics associated with the pediatric and adult clinical trials for NPB patients.  At this time, Genzyme is interested in receiving feedback from the patient community about the clinical trial.  If, after reviewing the information provided below, you should have a specific question about either clinical trial ~ please feel free to post them below in the comment section. 

The NNPDF Central Office will gather these, provide the list to Genzyme and then develop a “Q & A” web page to address all of your inquiries.  In the interim, should you need or require any additional assistance and or support, please feel free to contact me at:  nnpdf@nnpdf.org

Genzyme, A Sanofi Company, currently has a Phase 1/2 pediatric trial in process.  In addition, the current timeline indicates that the Phase 2/3 adult clinical trial began earlier this year will continue to enroll adult patients throughout 2016.

You can find more information regarding each trial below on the clinicaltrial.gov website:

Phase 1/2 pediatric trial

Phase 2/3 adult clinical trial

Genzyme has a brochure regarding the Acid Sphingomyelinase Deficiency (ASMD) Clinical Program that you can download at:

ASMD Clinical Program

The NNPDF also has a web page with an historical timeline of information associated with each trial here:  http://nnpdf.ca/npresearch_11.html

Chris Klauer
NNPDF Family Services and Marketing Coordinator
nnpdf@nnpdf.org

[Dec 3rd, 2015 ~ blg]

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It’s HERE ~ #GivingHOPETuesday ~ December 1st, 2015!  Today is the day that you can make a big impact on behalf of the National Niemann-Pick Disease Foundation, our Niemann-Pick Disease patients and the families we serve!  With your help, we’ll raise funds to assist with our NPD family services and research programs.

If you make a gift online TODAY, the foundation will earn matching funds from our partner, Network for Good—making your support go even further.

It’s easy to take part!


Donate Now button

Today’s the day, and we need your help to grow our impact for all NPD children and young adults living with Niemann-Pick Disease!

Heartfelt thanks!

NNPDF

[Dec 1st, 2015 ~ blg]

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Today is Cyber Monday when many will be shopping on-line for all the great holiday deals!  Before your friends and family get wrapped up in the online mania, please take a moment to remind them about the role that the NNPDF plays in your lives and how their donations to the foundation will be matched on Tuesday, December 1st, 2015!   

We've put together an e-News blast for you to share with friends and family showing some special opportunities available to you for #GivingHopeTuesday, which includes ways you can shop for the holidays and donate at the same time!

  • Matching funds from Network for Good for every donation made on December 1st at www.nnpdf.org
  • Bravelets donating double ($20 instead of $10) for all jewelry purchased on December 1st.
  • Holiday shopping options that donate back to the NNPDF a percentage of your purchase.

Click here to view the e-Blast: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1343158

[Nov 30th, 2015 ~ blg]

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Getting involved and promoting #GivingHOPETuesday is easy!  Here are a few simple ideas:

  • Consider making a gift on #GivingHOPETuesday at: https://donatenow.networkforgood.org/nnpdf?code=Homepage
  • Help us get the word out on social media, using @nnpdf and the hashtag #GivingHOPETuesday.
  • Take photos to post on Facebook, Twitter, or Instagram to show your support for of the National Niemann-Pick Disease Foundation.

With your help we can raise NPD Awareness and much needed research funds!

Donations made on Tuesday, December 1st through  #GivingHOPE Tuesday will help sponsor research and provide family support to the many NPD families that are members of the NNPDF.   By sharing this post you can help provide HOPE to so many in the NPD Community.  Go to www.nnpdf.org and click on the “Donate Now” button.

Persist. Quest. Cure.

[Nov 29th, 2015 ~ blg]

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We’re raising funds on #GivingHOPETuesday to help kick-start our 2015 giving season in support of the critical programs, services and research offered to our NPD patients and their families through the work of the NNPDF.

Not only will the foundation benefit from the funds raised on #GivingHOPETuesday, but we will also earn matching funds from our partner, Network for Good, for gifts given online—Tuesday, December 1st, 2015 ~ making your support go even further.

We need your help, and it’s easy.

  • Please consider making your year-end gift on December 1. If you wish to make your gift go further with matching funds, give online at https://donatenow.networkforgood.org/nnpdf?code=Homepage.
  • Help us get as many people involved as possible by spreading the word.
  • Send an email to your friends, colleagues, and family members sharing your enthusiasm for the NNPDF and our program services. We’d love them to support the foundation on #GivingHOPETuesday, but we also want everyone to support whatever inspires them.

• Help us get the word out about the NNPDF on Facebook and Twitter.

Connect with us at https://twitter.com/nnpdf and use the hashtag: #GivingHOPETuesday

Please do what you can for the families of the NNPDF on this big day. Your generosity WILL have an even stronger impact with the power of #GivingHOPETuesday and with our access to matching funds. Heartfelt thanks for your consideration and generosity.  We WILL Perservere in our QUEST for a CURE!

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[Nov 28th, 2015 ~ blg]

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Your passion to support and impact the lives of our patients and family membership affected by Niemann-Pick Disease can go even further on #GivingHOPETuesday .

On December 1, the NNPDF will be joining a national day of generosity, #GivingHOPETuesday. It’s a day when you can make a big impact on the National Niemann-Pick Disease Foundation, our NPD patients and their families.

With your help, we’ll raise funds to continue on with NPD family services programs, support, and referrals, as well as, funding for research into Niemann-Pick Disease.

If you make a gift online this Tuesday, December 1st, the NNPDF will earn matching funds from our partner, Network for Good—making your support go even further.

Check in tomorrow to see how easy it is for you to take part!  In the interim, share this post and link with all of your family and friends on Social Media! 

https://donatenow.networkforgood.org/nnpdf?code=Homepage

Corbitt

[Nov 27th, 2015 ~ blg]

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2015 Season of Giving and Thanks!

HT

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation would like to send everyone wishes for a safe and happy Thanksgiving.

Time and again, our NPD families have shown that, despite the challenges faced, they give thanks for all the opportunities which arise from those struggles through Perseverance, as only NPD families can.

We are very thankful for your continued support and that you allow us into your lives, where we hold you close in our thoughts and our hearts year round.

Best wishes to all for a warm and love-filled Thanksgiving holiday,

~ The Board Members and Staff of the NNPDF

[Nov 26th, 2015 ~ blg]

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DOUBLE DONATION DAY!  Start your holiday shopping early!!  In honor of #GivingHOPETuesday, December 1st, 2015 ~ Bravelets is donating $20 (instead of $10) out of every purchase of all BRAVELETs made on Tuesday, December 1st, 2015!

To help support the programs of the NNPDF and check off items for your holiday gift list, simply visit the Brave Page of the NNPDF and choose a Bravelet design that best fits your style. Won’t you consider joining along with the NNPDF in helping us to give back to our NPD patient community?

Click the link below on 12/1/15 to start shopping and send DOUBLE DONATIONS to the NNPDF so that you too can give the gift of HOPE to our NPD patients and their families.

 

Bravelets Gift of Hope

3brave

      Still need to obtain your personalized FaceBook icon and cover page ~ there is still time?....E-mail Beth at:  bgreen@nnpdf.org

[Nov 25th, 2015 ~ blg]

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Have you shared your #GivingHOPETuesday FaceBook icon and banner highlighting your NPD loved ones photo with all of  your family and friends on FB?  We are encouraging each family of the NPD foundation to enlist the support of FIVE (5) of your nearest and  dearest to update their FB icons and banners with your child’s personalized #GivingHOPETuesday FB icons!  It’s not too late to get your FB banner to use in the days ahead as we count down to December 1st, 2015 as #GivingHOPETuesday! 

Please remind your supporters that they can offer the gift of HOPE by going to www.nnpdf.org and clicking on the “Donate Now” button!  To further highlight their support of your family and the NNPDF, they will receive an “I donated to the NNPDF” FB icon button to point out heartfelt efforts in giving HOPE to all NPD families.

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E-mail Beth at:  bgreen@nnpdf.org

[Nov 24th, 2015 ~ blg]

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November/December 2015 CCNNPDF e-Newsletter ~ Now Online

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Dear Niemann-Pick Disease Families and Friends,

The CCNNPDF November/December 2015 e-Newsletter is available ~ Highlights include:

  • Meeting of the International Niemann-Pick Disease Alliance
  • "Think Again. Think NPC"
  • October 2015 GLOBAL Niemann-Pick Disease Awareness Month
  • November is National Family Caregivers Month
  • Giving Tuesday ~ December 1st, 2015
  • PersevereWear ~ Getting Ready for the Holidays!
  • Clinical Trials: Updates & Recruitment
  • Reminder from Chris Klauer

Click here to view the CCNNPDF November/December 2015 e-Newsletter!

[Nov 24th, 2015 ~ blg]

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#GivingHOPETuesday is December 1st, 2015.  #GivingTuesday was developed by the philanthropic community to ensure that on the heels of Black Friday, Small Business Saturday and Cyber Monday those who wish to begin the Holiday Season in the “Spirit of Giving” have the opportunity to give the gift of HOPE to the charity of their choice!!  Your directed donations to the NNPDF have a direct impact on those diagnosed with and living courageously with NPD every day!  The NNPDF has adopted: #GivingHOPETuesday to remind everyone that their donations will provide the Gift of HOPE to our NPD patients, their families and loved ones this holiday season.

How can you help???  We have laid this out for you in THREE easy steps!

1. Contact Beth at:  bgreen@nnpdf.org with a photo of your NPD family member so that she can create your personalized #GivingHOPETuesday ~ Facebook icon and banner as shown below:

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Hope

2. Upload the personalized #GivingHOPETuesday FaceBook (FB) icons and banners to your personal FB pages and encourage your family and friends to do the same! 

SPECIAL CHALLENGE:

We would like to CHALLENGE each NPD family in our Niemann-Pick Disease Foundation membership to recruit FIVE (5) friends & family members to update their FB icons and cover pages with your special #GivingHOPETuesday graphics! Likewise, encourage them to contact their FB friends and assist with this effort.  Let’s all work together to blanket Social Media with the beautiful faces, smiles and spirit of all our precious loved ones fighting Niemann-Pick Disease! 

3. Watch for daily posts from the foundation during this special week of “Thanks and Giving” which will all lead up to #GivingHOPETuesday, on December 1st, 2015!  The more we all promote and share these updates with your supporters the better able we will be to raise awareness and funds in support of the NNPDF and our programs.

You can learn more about the origins of #GivingTuesday at:http://www.givingtuesday.org/about/  

Any questions ~ or ~ need some guidance….please feel free to contact Chris Klauer, NNPDF Family Services and Marketing Coordinator at:  cklauer@nnpdf.org or call:  1-877-287-3672.  

I am here to help!

Chris

[Nov 23rd, 2015 ~ blg]

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From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator

The end of the year is coming up fast!  I know your families will be busy with holiday season activities and family gatherings but please remember to take some time for yourself ~ especially as November is National Caregivers Month!

My update this week highlights:

~ #GivingHOPETuesday!
~12 Days, 12 Ways; Caring for the Caregiver
~ 51 & Done!

Visit the Family Services web page on the NNPDF website to view the update: http://www.nnpdf.org/FamilyServiceSupport.html#Nov2015

Please feel free to contact me at: nnpdf@nnpdf.org or give me a call at: 1-877-287-3672 if you have any questions pertaining to the work of the foundation.  If you find a great resource, please pass it along so I can share it with the rest of our NNPDF Family Membership!

Have a great weekend!

Chris Klauer

NNPDF Family Support and Marketing Coordinator

[November 20th, 2015 ~ blg]

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November 2015 e-Newsletter ~ Now Online

Banner

Dear NNPDF Families and Friends,

The NNPDF November 2015 e-Newsletter is available! Highlights include:

  • Meeting of the International Niemann-Pick Disease Alliance
  • October 2015 GLOBAL Niemann-Pick Disease Awareness Month
  • November is National Family Caregivers Month
  • Giving Tuesday ~ December 1st, 2015
  • PersevereWear ~ Getting Ready for the Holidays!
  • Clinical Trials: Updates & Recruitment
  • Reminder from Chris Klauer

Click here to view the NNPDF November 2015 e-Newsletter!

[Nov 17th, 2015 ~ blg]

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From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator

October NPD Awareness Month was a great success!  Everyone did their part in raising awareness for Niemann-Pick Disease!

My update this week highlights:

  • ~October Awareness ~ Review
  • ~National Family Caregivers Month
  • ~ Giving Tuesday

Visit the Family Services web page on the NNPDF website to view the update: http://www.nnpdf.org/FamilyServiceSupport.html#Nov1315

Please feel free to contact me at: nnpdf@nnpdf.org or give me a call at: 1-877-287-3672 if you have any questions pertaining to the work of the foundation.  If you find a great resource, please pass it along so I can share it with the rest of our NNPDF Family Membership!

Have a great weekend!

Chris Klauer
NNPDF Family Support and Marketing Coordinator

[November 13th, 2015 ~ blg]

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NPD Current Clinical Trial Recruiting Update ~ e-Blast

blast

Dear National Niemann-Pick Disease Foundation (NNPDF) Family Membership,

The NNPDF Central Office is working diligently to ensure that our entire NPD membership community is aware of the various clinical trial opportunities available for those seeking innovative therapeutic options for Niemann-Pick Disease. With this in mind, the foundation is providing an update and links to updates which we have received from the three pharmaceutical companies currently moving forward with clinical trial opportunities for NPD patients.

To view the e-news Blast with the updated information dated November 11th, 2015, click here

[Nov 11th, 2015 ~ blg]

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Just a reminder that the following NNPDF Fundraising and NPD Awareness Events are happening:

Tonight, Monday, November 9th in Latham, New York is The Wide World of Sports III ~ In Honor of JP Honsinger.

If you can support them it would be greatly appreciated!

KUDOS to Anne O’Connor Smith for participating in her 1st Ironman Triathlon in Florida this weekend!  She had to run, swim and bike to reach the finish line!  WAY TO GO ANNE!!!

If you would like to add your event to the NNPDF Events Page please contact Chris Klauer, Family Services & Marketing Coordinator at nnpdf@nnpdf.org!

During October Awareness Month the NNPDF Central Office received notification of events happening in various states!  If you raised awareness in your state during October please let us know!  We will add you to the map!

October Awareness Map

Please remember that if you are holding an event and need assistance we can help you with so many of the details.       

  • On-line event registration page
  • NNPDF Liability Insurance Coverage Certificate

There are certain restrictions so please check with the NNPDF Central Office as to how we can assist!

NNPDF Fundraising Services Plan

As always you can find all of the NNPDF Current Fundraising and NPD Awareness Events at http://www.nnpdf.ca/donorinfo_13.html#Current.

[Nov 9th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 31 ~ Fast Facts

To learn more about what a NPD patient and their families face when a loved one is diagnosed with NPD ~ please refer to: http://www.nnpdf.org/npdisease_19.html & http://npatimeline.blogspot.com/

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 31st, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 30 ~ Fast Facts

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You can see more information about last year's conference at: http://www.nnpdf.org/FCRecaps.html.

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 30th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 29 ~ Fast Facts

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Go to: http://www.nnpdf.ca/Newsline2015.html and http://www.nnpdf.ca/ASMDNewsline2015.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 29th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 28 ~ Fast Facts

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You can visit the two Facebook group pages here:

https://www.facebook.com/nnpdf.staff

https://www.facebook.com/groups/nnpdf/

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 28th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 27 ~ Fast Facts

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To learn more about the NNPDF Equipment Exchange Program go to: http://www.nnpdf.ca/familyservices_09.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 27th, 2015 ~ blg]

Div

October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 26 ~ Fast Facts

To learn more about the Diagnosis of NPD go to: http://www.nnpdf.org/npdisease_02.html.

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 26th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 25 ~ Fast Facts

All the fundraisers and awareness events are posted at http://www.nnpdf.ca/donorinfo_13.html.

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 25th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 24 ~ Fast Facts

To learn more go to: http://www.nnpdf.ca/aboutus_19.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 24th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 23 ~ Fast Facts

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To see our colored in map go to: http://www.nnpdf.org/GlobalOctoberNiemann-PickDiseaseAwarenessMonth2015.htm. Next year can you help fill it even more?

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 23rd, 2015 ~ blg]

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Vtesse, Inc. Expands Scientific Advisory Board and Appoints new VP of Clinical Operations to Support Late-Stage Clinical Study of Lead Drug Candidate VTS-270
Dateline: 10/22/2015

Vtesse

Dear NNPDF Type C Families and Friends,

The NNPDF Central Office is pleased to share with our NPC Community a Press Release from Vtesse regarding a new addition of Elizabeth Berry-Kravis, M.D., Ph.D. to the Scientific Advisory Board for Vtesse and Michael Massaro as the new Vice President of Clinical Operations.

These new additions to Vtesse will enhance the team that is working to advance the clinical study of VTS-270 for Niemann-Pick Disease Type C. 

Click here to view the full Press Release Vtesse dated October 22, 2015

[Oct 23rd, 2015 ~ blg]

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Anna Beck-Policoff (NPC)
02/08/1995 ~ 06/04/2015

Anna

Dear NNPDF Families and Friends,

It is with heavy hearts and great sadness that we notify our NPD family membership of the passing of Anna Beck-Policoff  (February 8, 1995 ~ June 4, 2015), who courageously fought the Niemann-Pick Disease Type C (NPC) battle for 20 years.

To Anna’s loving father, Stephen, and her cherished younger sister, Jane, please know that your NNPDF family is here for you and are holding you all in our hearts and thoughts. We are truly heartbroken for you all but are comforted in knowing that the love and support you receive from those who loved Anna most will provide you the fortitude and strength that you will require in the coming days, weeks and months ahead without your precious Anna at your sides. Please reach out to us if you need us ~ we will be here for you.

[Oct 23rd, 2015 ~ blg]

Div

Genzyme

Dear NPB Families,

At the 23rd Annual NNPDF Family Support and Medical Conference held in Chicago, Illinois, some individuals of the NPB community were able to take part in the Patient Reported Outcome.  Please follow the link below to read what participants in this Patient Reported Outcome had to say.  They are currently looking for caregivers of our pediatric NPB community to take part in this interview.  

Click here to view the full post: http://www.nnpdf.ca/npresearch_11.html#Genzyme10222015

Thank you for your help!

[Oct 22nd, 2015 ~ blg]

Div

October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 22 ~ Fast Facts

22

To see some great ideas to go: http://www.graphicmail.com/new/viewnewsletter2.aspx?SiteID=4117&SID=0&NewsletterID=1332807

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 22nd, 2015 ~ blg]

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October 2015 e-Newsletter ~ Now Online

Banner

Dear NNPDF Families and Friends,

The NNPDF October 2015 e-Newsletter is available! Highlights include:

  • Global October Awareness Month
  • NNPDF 2015 Family Conference ~ Update
  • Clinical Trials: Updates & Recruitment
  • NNPDF Fellowship Progress Reports

Click here to view the NNPDF October 2015 e-Newsletter!

[Oct 21st, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 21 ~ Fast Facts

21

To learn more about the members of the INPDA go to: http://inpda.org/about-us/#content-area-2

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 21st, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 20 ~ Fast Facts

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To learn more about the INPDA please go to: http://inpda.org/about-us.

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 20th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 19 ~ Fast Facts

Day19

To learn more about the SAB Members please go to: http://www.nnpdf.org/aboutus_05.html.

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 19th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 18 ~ Fast Facts

Day18

To see the current and all past E-newsletters please go to http://www.nnpdf.ca/familyservices_04.html.

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 18th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 17 ~ Fast Facts

To become a member please go to: https://nnpdf.givezooks.com/events/national-niemann-pick-disease-foundation-membership-for

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 17th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 16 ~ Fast Facts

Fact16

To learn more about the research that is funded by the NNPDF and its supporters go to http://www.nnpdf.org/npresearch_04.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 16th, 2015 ~ blg]

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2015 GLOBAL Niemann-Pick Disease Awareness Month ~ Awareness e-Blast
Dateline: 10/16/2015

Global Niemann-Pick Disease Awareness Month has started and you are all doing a great job of raising awareness.  The NNPDF Central Office wanted to be sure that there are ways for everyone to take part in this event, so we put together an e-Blast full of fun and easy ideas.

For more information on things you can do to help raise awareness this October, click here.

[Oct 16th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 15 ~ Fast Facts

CAN

To learn more about The Canadian Chapter of the National Niemann-Pick Disease Foundation please go to http://nnpdf.ca/aboutus_03.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 15th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 14 ~ Fast Facts

Spencer

We have gathered information highlighting what participating in a clinical trial can mean for your NPD family member,as well as resources to support your family in making the decision that is right for all involved, please go to http://www.nnpdf.org/ClinicalTrialFAQ.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 14th, 2015 ~ blg]

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UPDATE FOR National Niemann-Pick Disease Foundation (NNPDF) NPC patient/family membership! ~ Clinical Trial Updates  for Niemann-Pick Disease Type C ~
Dateline: 10/12/2015

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Attn:  NNPDF NPC Family Membership

One of the key aspects of the NNPDF of the program services and research strategy which is included within the foundations goals and objectives is to ensure that our patient community is kept informed as to upcoming therapeutic and clinical trial opportunities.

In addition, the foundation works to ensure that all involved have access to the tools and information necessary to garner a solid understanding of the clinical trial itself, as well as the process of your loved one taking part in the trial.  With this in mind, the NNPDF is sharing the following correspondence associated with the Vtesse VTS-270 clyclodextrin clinical trial.

Should you have any questions regarding this information, please feel free to contact the NNPDF Central Office or a member of the “51 & Done!” Parent committee.

To read the full letter dated 10/12/2015, please visit the NNPDF "51 and Done!" web page.

[Oct 14th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 13 ~ Fast Facts

13

To watch this short film which was funded and supported by Hope for Hollie Foundation, Niemann-Pick UK and the International Niemann-Pick Disease Alliance go to https://www.youtube.com/watch?t=60&v=4gVcI_0T5l4

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 13th, 2015 ~ blg]

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Jaymee Emma Hernandez (NPC)
05/07/2011 ~ 10/13/2015

Hernandez Family

May 7, 2011 - October 13, 2015 ~ NPC, Age 4 Years

Dear NNPDF Families and Friends,

It is with heartbreak and sadness that we notify our NPD family membership of the passing of Jaymee Emma Hernandez (May 7, 2011 ~ October 13, 2015), who courageously fought the Niemann-Pick Disease Type C (NPC) battle for 4 years.

Jaymee just celebrated her 4th birthday with her family and friends.  She had a wonderful Curious George themed birthday party.  She had just attended her first day of Pre-K on September 3rd.   It was an exciting day and a huge milestone for Jaymee and her family.

To Jaymee’s loving parents, Jessica and Pedro, and her beloved older sister, Malory, please know that your NNPDF family is here for you and are holding you all in our hearts and thoughts.  We are truly heartbroken for you all but are comforted in knowing that the love and support you receive from those who loved Jaymee most will provide you the fortitude and strength that you will require in the coming days, weeks and months ahead without your precious Jaymee at your sides.  Please reach out to us if you need us ~ we will be here for you.

When Memorial Service information and how to send condolences to the family becomes available, we will update this post.

[Oct 13th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 12 ~ Fast Facts

To learn more about the latest news and updates pertaining to Clinical Trials please go to: http://www.nnpdf.org/#ClinicalTrials

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 12th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 11 ~ Fast Facts

To learn more about how the NNPDF serves our foundation family membership, please follow this link with highlights from the NNPDF Family Services and Marketing Coordinator, Chris Klauer:http://www.nnpdf.org/FamilyServiceSupport.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 11th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 10 ~ Fast Facts

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To learn more about PersevereWear and other NPD Promotional Products, please follow this link:http://www.nnpdf.ca/store.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 10th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 9 ~ Fast Facts

To learn more about the role of a NNPDF Board Member, please follow this link: http://www.nnpdf.org/aboutus_03.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 9th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 8 ~ Fast Facts

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You can learn more about Niemann-Pick Disease Type C by following this link : http://www.nnpdf.ca/npdisease_01.html#NPC

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 8th, 2015 ~ blg]

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October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 6 ~ Fast Facts

Gregorio

To learn more about Niemann-Pick Disease Type A (NPA / ASMD) please follow this link:

http://www.nnpdf.ca/npdisease_01.html#ASMD

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 6th, 2015 ~ blg]

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Bio Report Podcast Featuring Ben Machielse from Vtesse, Inc.
Dateline: 10/06/2015

Vtesse

Dear NNPDF Type C Families and Friends,

Last week, Ben Machielse, President and CEO at Vtesse, had an opportunity to sit down with Daniel Levine for a segment featured on the Bio Report Podcast.   The Bio Report Podcast focuses on the intersection of biotechnology with business, science, and policy.

Ben was able to focus on the progress made in their clinical development program associated with the Phase II/III for VTS-270 a modified form of cyclodextrin.   We are sharing the link for you to listen to the audio recording of the pod cast below:

Rare Disease Drug Developer Shows Speed of Business Model

[Oct 6th, 2015 ~ blg]

Div

October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 5 ~ Fast Facts

Zoe Trina
Mason

To learn more about the inheritance factor associated with NPD and how genetics plays a part please go to the NNPDF web links below:

Niemann-Pick Type A: http://www.nnpdf.ca/npdisease_11.html
Niemann-Pick Type B: http://www.nnpdf.ca/npdisease_12.html
Niemann-Pick Type C: http://www.nnpdf.ca/npdisease_13.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 5th, 2015 ~ blg]

Div

October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 4 ~ Fast Facts

For more information on NNPDF current fundraisers and NPD Awareness events, visit: 
http://www.nnpdf.ca/donorinfo_13.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 4th, 2015 ~ blg]

Div

October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 3 ~ Fast Facts

For information on ALL types of Niemann-Pick Disease, visit:
http://www.nnpdf.org/npdisease_01.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 3rd, 2015 ~ blg]

Div

October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 2 ~ Fast Facts

D2

For information on ALL types of Niemann-Pick Disease, visit: http://www.nnpdf.ca/npdisease_01.html

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 2nd, 2015 ~ blg]

Div

October 2015 ~ GLOBAL ~ Niemann-Pick Disease Awareness Month
Day 1 ~ Fast Facts

Day1

For information and additional tools on how YOU can help raise awareness this month for Niemann-Pick Disease Month, visit the 2015 October GLOBAL Niemann-Pick Disease Awareness Month Page.

For a quick review of all the posts follow this link: http://www.nnpdf.org/2015OctAwarenessDailyPosts.html

[Oct 1st, 2015 ~ blg]

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Banner

In preparation for Global October Niemann-Pick Disease Awareness month, the NNPDF Central Office has some great ways to get you, your extended family and community involved in raising awareness for NPD!

For all of our families hosting FR & Awareness events ~ we encourage our family membership to reach out with words of encouragement!  Better yet, if you know of family & friends who live nearby ~ notify them of the event!  The more the merrier!

UPDATE:  We WANT your photos!  The NNPDF will once again be posting the “NPD Fact of the Day” every day in October to help with awareness!  We will utilize photos of ALL our NPD loved ones as we develop these daily posts.  If you are interested in having your loved one’s photo to be included on one of the “Fact Posts” please e-mail them to:  nnpdf@nnpdf.org   For more details see my weekly post, as well as the following topics:

  1. Upcoming Fundraisers ~ this week!
  2. Custom Facebook Cover Photos & Icon Graphics (let us do the work for you!! )
  3. NPD Facts of the Day ~ Calling for all NPD children/young adult photos!

Follow this link for updates on these and other topics: http://www.nnpdf.org/donorinfo_13.html#Sep282015

[Sep 28th, 2015 ~ blg]

Div

Vtesse, Inc. Initiates Phase 2b/3 Clinical Trial of VTS-270 for Treatment of
Niemann-Pick Type C1 (NPC) Disease

Vtesse

Dear NNPDF NPC Patient and Family Community~

The National Niemann-Pick Disease Foundation (NNPDF) is pleased to advise our Niemann-Pick Disease Type C patient & family community that Vtesse, has received FDA authorization to begin recruiting for the Cyclodextrin / VTS-270 clinical trial for patients in the United States, diagnosed with NPC1, who fall within the age range of 6 to 21 years.

Please click below to access the letter and press release!

Vtesse Press Release is at: http://www.nnpdf.org/documents/092815Vtesse_Ph2_3trialstartreleaseFINAL_Complete.pdf

You can find all information regarding historical information about Vtesse and the VTS-270 clinical trial at http://www.nnpdf.ca/Vtesse.html.

If you have any questions, as always, contact the NNPDF Central Office at: nnpdf@nnpdf.org.

[Sept 28th, 2015 ~ blg]

Div

September 2015 e-Newsletter ~ Now Online

Banner

Dear NNPDF Families and Friends,

The NNPDF September 2015 e-Newsletter is available! Highlights include:

  • NNPDF 2015 Family Conference Recap
  • Clinical Trials: Updates & Recruitment 
  • Global October Awareness Month
  • Reminders from Chris Klauer, Family Services & Marketing Coordinator

Click here to view the NNPDF September 2015 e-Newsletter!

[Sept 15th, 2015 ~ blg]

Div

Rachel Noel Palmiter (NPC)
12/20/1984 - 08/28/2015

Rachel

Rachel Noel Palmiter, December 20, 1984 - August 28, 2015 ~ NPC, Age 30 years

Dear NNPDF Families and Friends,

It is with a sorrowful heart that we notify our NPD family membership of the passing of Rachel Noel Palmiter (NPC, age 30 years), who courageously battled Niemann-Pick Disease.

As a young adult diagnosed with Niemann-Pick Disease, Rachel’s parents, Wayne & Lori worked diligently to ensure that she kept her independence as a young woman.  Rachel cherished her friendships with the individuals that she lived with. 

To Rachel’s loving parents, Wayne and Lori, and her brother, Ryan (NPC), please know that your entire NNPDF family is here for you and holding you all in our hearts and thoughts.  Please know that we are thinking of you as you walk this path without your cherished Rachel at your side in the days, weeks and months ahead.

For Memorial Service information and how to send condolences to the family: Click here

[Sept 1st, 2015 ~ blg]

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"For a Rare Disease, Drug Trials Scramble for Patients"
A Wall Street Journal Article
By Amy Marcus

WSJ

Dear NNPDF Families and Friends,

Last night an article written by Amy Marcus of the Wall Street Journal was published. This article discusses the fact that there are three possible clinical trials on the horizon for our Niemann-Pick Type C patient community and that the need for patient recruitment and community support is critical.

Wall Street Journal Article ~ http://www.wsj.com/articles/for-a-rare-disease-drug-trials-scramble-for-patients-1440013683

This article discusses the plan to recruit 51 patients at 20 sites around the world for the trial that Vtesse, Inc. is moving forward with in regards to cyclodextrin. Recently the NNPDF sent out correspondence to all NPC families regarding the “51 and Done!” campaign. The letter asked for all families to sign on and show support for this trial.

You can find a copy of these documents at this link: http://www.nnpdf.org/documents/FullPackettoCommunity.pdf

If you have questions please feel free to contact us at: nnpdf@nnpdf.org.

We will PERSEVERE for a cure!

Nadine Hill
Executive Director

[Aug 20th, 2015 ~ blg]

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AUGUST 2015 e-Newsletter ~ Now Online

Banner

Dear NNPDF Families and Friends,

The NNPDF AUGUST 2015 e-Newsletter is available! Highlights include:

  • NNPDF 2015 Family Conference Recap
  • Equipment Exchange Update
  • Research & Clinical Trial Updates
  • Reminders from Chris Klauer, Family Services & Marketing Coordinator

Click here to view the NNPDF August 2015 e-Newsletter!

[Aug 18th, 2015 ~ blg]

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Katherine Grace “Katie Grace” Limer (NPC)
08/06/2005 ~ 8/11/2015

Katie
August 6, 2005 – August 11, 2015 ~ NPC, Age 10 years

Dear NNPDF Families and Friends,

It is with a sorrowful hearts that we notify our NPD family membership of the passing of Katherine Grace Limer (August 6, 2005 – August 11, 2015), who courageously fought the NPC battle for 10 years.

Katie Grace just celebrated her 10th birthday with her family and friends in a beautiful white dress.  Her cake was colorful just like her personality.  She loved listening to “Big Time Rush” and singing out loud often to the rooftops!

Many in our NNPDF family membership will recall the day that Katherine Grace “Katie” was born as she made her special appearance into this world ~ while her NPD families were gathered at the Manhattan Beach, California Family Conference in 2005!  We all celebrated with great joy at her arrival!

Katie and her family last joined us at the 2012 Family Conference in Nashville, TN.  She shared her sweet sparkle of a smile and twinkle in her eye with all that she met and joined us for the Saturday evening gala & dance dressed in bedazzled in red sparkles, a silver glitter gloved hand and hat to top off her gleaming attire!

Katie Grace’s family note that she is now gleefully playing in heaven with her sister, Rebecca Marye and brother, Andrew Thad, who also suffered from Niemann-Pick Disease and who passed away in 2004 and 2005 respectively.

To Katie Grace’s loving parents, Thad and Leah, and her beloved older sister, Megan Elizabeth, please know that your NNPDF family is here for you and holding you all in our hearts and thoughts.  We are truly heartbroken for you all but are comforted in knowing that the love and support you receive from those who loved Katie most will provide you the fortitude and strength that you will require in the coming days, weeks and months ahead without your precious Katie at your sides.  Please reach out to us if you need us ~ we will be here for you.

We have learned that Katie’s visitation & services will be held at: Wade Funeral Home; 4140 W. Pioneer Parkway ; Arlington, Texas 76013.  Visitation will be on Friday, August 14th, 2015 from 6pm – 8pm with services on Saturday, August 15th at 11:00am. http://www.wadefamilyfuneralhome.com/

Here is a news video which highlights the Limer family in 2008 when Katie was just 3 years old ~ sweet memories: https://youtu.be/qdFXqDKhAQc

Click here read her obituary and leave condolences for the family.


[Aug 12th, 2015 ~ blg ~ UPDATED| Aug 14th, 2015]

 

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Vtesse, Inc. Announces Preliminary Data from Ongoing Phase 1 Study of VTS-270 for Treatment of Niemann-Pick Disease Type C

Vtesse

The NNPDF Central Offices are pleased to share with our NPC community that Vtesse, Inc. announced preliminary results today from an open-label Phase 1 clinical trial with VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for treatment of Niemann-Pick Disease Type C (NPC) conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

Preliminary analyses, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardized measure) in children treated with VTS-270 in the Phase 1 trial as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients. The analyses also show that children treated with VTS-270 demonstrated improvement on several disease domains.

To view the full press release, as well as the archival history of this clinical trial, please visit the NNPDF Vtesse web page.


[Aug 6th, 2015 ~ blg]

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Genzyme logo

 

Update from Genzyme on Acid Sphingomyelinase Deficiency (ASMD)
Development Efforts

US Investigational Site Now Open for Recruitment
August 6th, 2015

 

ALERT: United States NPD Type B Families & Friends

The NNPDF central office noted today that the first investigational site for Genzyme's Acid Sphingomyelinase Deficiency (ASMD) Pediatric Trial has been updated and is now actively recruiting pediatric patients at the approved clinical trial center in New York, NY.

Families should follow enrollment criteria as provided on the www.clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit www.clinicaltrials.gov and refer to study reference number: NCT02292654

For additional information, as well as, all previous updates regarding this Genzyme's ASMD clinical trials, visit the NNPDF Genzyme Clinical Trial webpage.

[Aug 6th, 2015 ~ blg]

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2015 Family Conference ~ Events, Travel Tips & Tid-bits e-Blast!

FC

Dear NNPDF Families and Friends,

In preparation for this week's conference, we've put together an e-Blast for our attendees to help you anticipate all that the conference has in store, as well as, some tips and tid-bits to make your time in Chicago fun and easy for everyone!

Click here to view the 2015 NNPDF Family Conference e-Blast!

If you have any questions about the conference or the information contained in the attached “e-news blast” ~ please feel free to contact the NNPDF Central Offices at: nnpdf@nnpdf.org or 1-877-287-3672.

Looking forward to welcoming you all to the “Windy City” on Thursday ~ “Where the Winds of Hope are on the Horizon!

Safe travels,
Nadine

[Aug 3rd, 2015 ~ blg]

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JULY 2015 e-Newsletter ~ Now Online

Banner

Dear NNPDF Families and Friends,

The NNPDF July 2015 e-Newsletter is available! Highlights include:

  • 23rd Annual NNPDF Family Support and Medical Conference
  • NNPDF Equipment Exchange Update
  • Research & Clinical Trial Updates
  • Reminder from Chris Klauer, Family Services & Marketing Coordinator
  • 2015 NNPDF Board Meeting ~ at the 23rd Annual Family Conference

Click here to view the NNPDF June 2015 e-Newsletter!

[July 22nd, 2015 ~ blg]

Div

cap

Hello NNPDF Families and Friends,

The NNPDF is pleased to announce to our Niemann-Pick Type B (ASMD) patient community a new “Qualitative Research Phase” titled:  Patient Reported Outcome (PRO) sponsored by Genzyme.  Patient-Reported-Outcome (PRO) instruments are measures self-reported by patients, about disease symptoms and impact, as well as impact of treatment.  Please review the attached announcement which further details the patient interview study and the essential component this information plays in support of the entire ASMD community.

In addition, the NNPDF has been able to work collaboratively with representatives from Genzyme and Evidera (the research consulting firm engaged to oversee this project) will be on-site to conduct “face-to-face” family and patient interviews at the upcoming 23rd Annual NNPDF Family Support and Medical Conference to be held in Chicago, Illinois ~ Thursday, August 6th thru Sunday, August 9th, 2015. 

Please follow this link to read the full Genzyme announcement:  Click here to read the full press release

For more information pertaining to the NNPDF Family Conference:  http://nnpdf.org/familyservices_03.html

[Jul 22nd, 2015 ~ blg]

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Webinar

Hello NNPDF Families and Friends,

We are delighted to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse's leadership and clinical team. As mentioned in the initial announcement, Vtesse is committed to keeping the community fully informed and soliciting feedback as we move toward the clinical trial process.

Two webinars will be held on July 14, 2015. As a participant, you will be able to ask any questions you may have and gain an understanding of:

• Recent developments at FDA and EMA
• Phase 1 data review
• Final Protocol including the inclusion and exclusion criteria
• Introduction of the Clinical Trial Website
• Update on the Fight NPC Patient Map

Please follow these links to the Vtesse “GoToMeeting” site to register for the calls:

Register for July 14th, 1:30pm EDT (UK - 6:30pm (BST):
https://attendee.gotowebinar.com/register/7192330336175692801

Register for July 14th, 7:30pm EDT (UK 12:30am, July 15th, BST): https://attendee.gotowebinar.com/register/7756655778643496705

For those who aren’t able to join the call ~ please be advised that Vtesse WILL be recording the sessions and making them available at a later date.

Vtesse is also welcoming any questions you may have prior to the webinar. You may send any correspondence to Carrie Burke at: carrie@vtessepharma.com.

Kind regards,
Nadine Hill
NNPDF Executive Director

[Jul 10th, 2015 ~ blg]

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F

Here at the NNPDF we appreciate all of those that are working together raising funds to help support our programs and promote research to find a cure for all of those affected by NPD.

Updates this week highlight the following topics:

  • NNPDF Promotional Products ~ Bravelets
  • NNPDF “Recipes for Success” ~ Home Party Fundraisers
  • NNPDF Awareness Posters

To read more on ways in which the NNPDF is able to assist you with your NPD fundraising and awareness events and campaigns ~ follow this link….

Visit the NNPDF Fundraising page to view the full post

We WILL Persevere in our Quest for a Cure!

 

[Jul 6th, 2015 ~ nmh]

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From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator

In support of all in our NPD patient & family membership community, I will be posting weekly informational updates and NNPDF related resource opportunities.  These posts will cover a broad range of topics and I welcome you to share any resources which you have identified and/or contact me if you need specific assistance with an issue.

This week I thought it would be nice to concentrate on the upcoming Holiday!  The 4th of July is a time when families do a great many activities as they spend time with family and friends.   This post has a story board, fun activities and reminders on how to avoid overload!

As the NPD community receives more information on experimental therapies and opportunities for involvement in clinical trials, I thought it timely to share an important NNPDF resource web page and article titled:  “Thinking About Experimental Therapies”, which offers tools to assist your family in addressing this vital decision. 

Preparations for the upcoming 23rd Annual Family Support and Medical Conference continue and the foundation has lined up an exceptional list of speakers for this event.  In addition, we are pleased to announce that the Loews Chicago O’Hare Hotel site choice was recently named one of the TOP 11 Airport hotels around the world by USA Today! 

Please contact me at: nnpdf@nnpdf.org or give me a jingle at: 1-877-287-3672 if you have any questions pertaining to the work of the foundation.  If you find a great resource, please pass it along so I can share it with the rest of our NNPDF Family Membership!

Have a great weekend!

To read the full update, visit the Family Support & Updates page.

Chris Klauer
NNPDF Family Support and Marketing Coordinator

[Jul 2nd, 2015 ~ blg]

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JUNE/JULY 2015 CCNNPDF e-Newsletter ~ Now Online

news

Dear Niemann-Pick Disease Families and Friends,

The CCNNPDF June/July 2015 e-Newsletter is available ~ Highlights include:

  • The 23rd Annual NNPDF Family Support and Medical Conference
  • Message from the NNPDF Board of Directors
  • APMRF Conference
  • CCNNPDF Membership ~ Action Required 
  • INPDA New Website
  • Clinical Trial News and Updates

Click here to view the CCNNPDF June/July 2015 e-Newsletter!

[July 1st, 2015 ~ blg]

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Vtesse

RE:  Vtesse NPC Community Town Hall Webinar ~ July 14th, 2015

Vtesse invites you to hear about their progress, ask questions and provide feedback

Dear NNPDF Niemann-Pick Type C (NPC) Family Members,

The NNPDF is delighted to be able to share with you an opportunity to attend a town hall webinar hosted by Vtesse’s company leadership and clinical team. As mentioned in their initial announcement, dated January 7th, 2015, Vtesse is committed to keeping the community fully informed and soliciting feedback as they move toward the clinical trial process for VTS-270 (cyclodextrin) for Niemann-Pick Disease, Type C (NPC).

This is your opportunity to be part of that discussion!

Two webinars will be held on July 14th, 2015. As a participant, you will be able to ask any questions you may have and gain an understanding of:

  • Recent developments at Food and Drug Administration (FDA) and European Medicines Agency (EMA)
  • Phase 1 data review
  • Protocol: inclusion and exclusion criteria
  • Introduction of the Clinical Trial Website
  • Update on the Fight NPC Patient Map

Please note: There is no cost to participate in this webinar and Vtesse welcomes any questions which you may have prior to the webinar. This webinar is for NPC patients worldwide and is currently set-up to accommodate families and participants from 18 countries. You are encouraged to pre-register and submit your questions to Vtesse via the "GoToWebinar" registration site listed below:

Register for July 14th, 1:30pm EDT (UK - 6:30pm (BST):
https://attendee.gotowebinar.com/register/7192330336175692801

Register for July 14th, 7:30pm EDT (UK 12:30am, July 15th, BST): https://attendee.gotowebinar.com/register/7756655778643496705

For our family membership in the United States and Canada who do not have access to a computer,  or require assistance in registering, please call the NNPDF Central Offices toll free at: 1-877-287-3672.  The NNPDF Central Office Staff will be more than happy to assist you in registering for the call and providing you with a telephone call-in number.   

The NNPDF looks forward to participating in this webinar on behalf of all our community membership and we hope you will take advantage of this opportunity as well. Please feel free to reach out to the NNPDF Office Staff at nnpdf@nnpdf.org if you have any questions.

We WILL PERSEVERE in our Quest for a Cure!

What is a Webinar?

A webinar is sometimes also referred to as a “webcast”, “online event” or “web seminar”

Webinars typically have audio and visual components and enable the sharing of PowerPoint presentations, videos, web pages or other multimedia content with audiences that can be located anywhere. The visual component of a webinar is shared through a web conferencing tool or Internet browser. The audio portion of a webinar is usually broadcast through the audience’s computers (through speakers and media players) or through the telephone.

[Jun 30th, 2015 ~ blg]

Div

 

Research Publication:
Successful Within-patient Dose Escalation of
Olipudase Alfa in Acid Sphingomyelinase Deficiency (ASMD)

Wass

The NNPDF Central received notification of a recent on-line publication highlighting the work of Dr. Melissa Wasserstein, and the invaluable work that she has been doing to develop a potential therapy for acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease Types A and B).

Dr. Wasserstein's work has been co-sponsored by the National Niemann-Pick Disease Foundation (NNPDF) and the Canadian Chapter of the NNPDF (CCNNPDF) since October of 2014.

An article was published in Elsevier's Molecular Genetics and Metabolism magazine online that we wanted to share with the ASMD community.

Click here to read the full article:
Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency

Follow this link to learn more about Dr. Wasserstein's specific research:  http://nnpdf.org/npresearch_05.html

[Jun 25th, 2015 ~ blg]

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Update on Niemann-Pick Disease, Type C

PR

Dear NNPDF Families and Friends,

The NNPDF Central Office is pleased to share with our NPC community the following press release.

Calporta Therapeutics is developing small molecule agonists of TRPML1 for the treatment of Niemann-Pick Disease, type C (NPC) and other lysosomal storage diseases. Calporta's approach is based on research from the lab of Haoxing Xu, Ph.D., at the University of Michigan.

Click here to read the full press release

As more information becomes available we will continue to update this website, as well as, our social media sites.

[Jun 23rd, 2015 ~ blg]

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From the Desk of Chris Klauer, NNPDF Family Services & Marketing Coordinator

In support of all in our NPD patient & family membership community, I will be posting weekly informational updates and NNPDF related resource opportunities.  These posts will cover a broad range of topics and I welcome you to share any resources which you have identified and/or contact me if you need specific assistance with an issue.

One thing I have learned from my many years of teaching students with special needs is that behind every child or adult is an awesome parent or caregiver supporting and addressing their every need.  Therefore, my post this week focuses on “Caring for the Caregiver” and Respite Care.  I have also included a quick “tid-bit” about the upcoming Chicago Family Conference.  Please review the NNPDF Family Services web page for my weekly posts and links to resources.

You can always reach me at: nnpdf@nnpdf.org or call me at 1-877-287-3672. I look forward to the opportunity to help you find resources related to your situation/issue and welcome hearing of the “tricks-of-the-trade” that you have learned and identified in your journey.

Have a great weekend!

~ Chris

[Jun 19th, 2015 ~ cdk]

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Bart Vernon Mackie (NPC)
05/31/1962 - 05/28/2015

Bart

Dear NNPDF Families and Friends,

It is with deep heartbreak that the NNPDF Central office passes along word that Bart Vernon Mackie (aged 52 years) has lost his courageous and long fought battle with Niemann-Pick Disease type C (NPC) on May 28, 2015.  Bart loved and played baseball in his twenties and later worked as a heavy equipment operator.

Bart’s family and friends will join together in loving celebration of his life on Saturday, June 20th at Noon at Hope Church at Silver Lake in Everett, Washington.  His family has generously asked that remembrances and memorials be made in Bart’s name to the National Niemann-Pick Disease Foundation at www.nnpdf.org .  Bart’s older brother Bradley G. Mackie preceded him in death on July 31st, 2014 at the age of 54 years from complications of NPC.

To Bart’s loving wife, Elaine of 26 years; his two beautiful and cherished daughters, Shanna and Kelsey; his mother Lynne and stepfather Jim Lambright; his father Vern and his sister Regan ~ we send our sincerest condolences and our hearts grieve along with you.  Please know that your NNPDF family is here for you as you travel down this path without your beloved son, brother, husband, father and friend at your side ~ not only today but in the days, weeks and months ahead.

To view the obituary and send your condolances to the family: Click here

{Jun 18th, 2015 ~ blg]

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A Message from Becky McGuire, NNPDF Development Chair
Dateline: June 8th, 2015

Becky McGuire Kelly
Becky McGuire
Cousin of...
... Kelly Thompson
NPC

Dear NNPDF Families and Friends,

New for 2015, the NNPDF Board of Directors rolled out a new communication strategy which we hope will assist you in feeling more connected with the work of the foundation and your Board Representatives. This new communication strategy offers the foundation a unique opportunity to share on-going efforts, as well as new and existing programs, projects and activities being addressed on your behalf, our family membership and NNPDF key stakeholder’s. Crucial to the role of the foundation is that of “communication liaison” between pharmaceutical and research agencies working to bring innovative therapies to our NPD patient community via clinical trials.

Today, we wish to share with you an update from the 2015 Development Committee Chair, Becky McGuire, of Torrington, Connecticut. Becky serves on the foundation board in a volunteer capacity and in honor of her cousin, Kelly Thompson (NPC, age 36 years).

Dev

The NNPDF Board of Directors (BoDs) wrapped up our annual board meeting in early March 2015 in Chicago, Illinois.  On Tuesday, March 24th, the first BoDs update was penned by the sitting 2015 NNPDF Board Chair, Leslie Hughes, which was followed by the Nominations Board Chair, Lisa Chavez's post on March 31st, 2015.  On April 16th, 2015 the foundation Finance Chair, Jill Flinton, shared her background and updates on the financial condition and efforts of the NNPDF.  Please find “quick links” to all of these previous posts below.

Click here to view Leslie Hughes, Board Chair's Message to the Community
Dateline March 24th, 2015

Click here to view Lisa Chavez, Nomination Chair's Message to the Community
Dateline: March 31st, 2015

Click here to view Jill Flinton, Finance Chair's Message to the Community
Dateline: April 16th, 2015

Click here to view Becky McGuire, Development Chair's Message to the Community
Dateline: June 8th, 2015

If you should have any questions related to this information and/or update ~ please feel free to contact the foundation for assistance.

[June 8th, 2015 ~ blg]

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Logo

 

NIH Pharmacy Development Section Issue-NOT applicable to NPC trials-notes Dr. Porter of NIH
NIH Press Release
Dateline: 06/04/2015

 


Dear NNPDF Families and Friends,

This afternoon a press release from the National Institutes of Health (NIH) outlined the discovery of a serious manufacturing problems and lack of compliance with standard operating procedures discovered during a recent  at the NIH Pharmacy  Development Section which the FDA inspected in late May 2015.  We have received the following comments from Dr. Forbes Porter as it relates to the two Niemann-Pick Disease Type C (NPC) clinical trials which are currently being conducted at the NIH.

MEMO

From: Dr. Forbes Porter
RE:
NIH Pharmacy Development Press Release
Dated:
Thursday, June 4th, 2015

To all:

I would like to notify you of an issue that has occurred in the NIH Pharmacy Development Section that is likely to hit the news.  Please see below.   I am concerned that there may be some confusion with respect to an investigational drug manufactured by the Pharmacy Development Section (PDS) and an investigational drug dispensed by the NIH pharmacy.

This does not affect either the cyclodextrin or vorinostat studies. 

We are currently in the process of  trying to notify the families that are participating in our trials.   Please feel free to post or distribute to those that may hear the news and have questions. 

Denny

Forbes D. Porter, MD, PhD
Senior Investigator, PDEGEN, NICHD, NIH
Program Head, PDEGEN, NICHD, NIH
Clinical Director, NICHD, NIH

Please find a copy of the full press release below.

Kind regards,
Nadine Hill
NNPDF Executive Director

~ For Immediate Release: Thursday, June 04, 2015 ~

NIH suspends operations in its Clinical Center Pharmaceutical Development Section
http://www.nih.gov/news/health/jun2015/nih-04.htm

The National Institutes of Health (NIH) Clinical Center has suspended operations of its Pharmaceutical Development Section (PDS) due to the discovery of serious manufacturing problems and lack of compliance with standard operating procedures. Upon receipt of a complaint, Food and Drug Administration (FDA) representatives inspected the PDS between May 19 and May 29, and found a series of deficiencies that will require the NIH Clinical Center to take a number of corrective actions.

The facility makes products for certain clinical research studies conducted in the hospital and collaborating facilities. In April, two vials of albumin, used for the administration of the drug interleukin in experimental studies, were found to have fungal contamination. Vials made from the same batch were administered to six patients, although it is unknown whether those or other vials were contaminated. The six patients have been notified and are being followed closely for any signs of infection. At this time, none has developed signs of infection or illness.

“This is a distressing and unacceptable situation,” said NIH Director Francis S. Collins, M.D., Ph.D. “The fact that patients may have been put in harm’s way because of a failure to follow standard operating procedures in the NIH Clinical Center’s Pharmaceutical Development Section is deeply troubling. I will personally oversee the steps to protect the safety of patients and remedy the situation as swiftly as possible.”

Among the problems the FDA identified in their inspection were deficiencies in the physical facility, including flaws in the air handling system, and operational failures including inadequate quality control, insufficient employee training, and lack of compliance with standard operating procedures. Deficiencies of lesser significance were identified in the Clinical Center Pharmacy. The FDA inspection reports are available here: http://www.cc.nih.gov/phar/pdfs/483.pdf (PDF - 1.31KB).

The following steps are being taken immediately to protect patients:

  1. Operations of The Pharmaceutical Development Section have been suspended and no products will be made or distributed until all problems are fully understood and corrected. Materials produced by the Section are being systematically tested for contamination.
  2. Of the participants in the 46 studies that are potentially affected, approximately 250 are currently scheduled to receive products manufactured by the PDS. NIH has notified the individual principal scientists responsible for each of those protocols, and is in the process of notifying the participants in these protocols. The vast majority of these patients are not immediately due for treatment and NIH is working to secure alternative sources for the products.
  3. An external group of experts in microbiology and sterile manufacturing practices will be appointed to conduct a thorough review, including an assessment of all standard operating procedures, policies, staffing, and training, and make recommendations to the NIH director on the corrective actions required.
  4. In addition to the immediate steps NIH is taking, it will provide an interim corrective action plan to the FDA by Friday, June 19, 2015.

“Our first responsibility is the safety and care of our patients,” said Dr. Collins. “NIH leadership is determined to identify and correct all of the deficiencies that have led to this situation.”

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.

Visit the NNPDF Cyclodextrin Page to view the full press release in PDF, and to see the full history of the Cyclodextrin clinical trial campaigns!

[Jun 4th, 2015 ~ blg]

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Genzyme logo

 

Update from Genzyme on
Acid Sphingomyelinase Deficiency (ASMD) Development Efforts
June 4th, 2015

 

Dear NNPDF Families and Friends,

The NNPDF Central Office is pleased to share a Press Release from Genzyme, a Sanofi Company, (dtd: Thursday, June 4th, 2015) which announces that the United States Food and Drug Administration (FDA) has granted “Breakthrough Therapy” designation to olipudase alfa. This enzyme replacement therapy (ERT) is being investigated for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick Disease type B.

“Breakthrough Therapy” designation is intended to expedite the development and review of investigational new drugs that target serious or life-threatening conditions which have an unmet therapy or medical treatment.

This is the FIRST time that Genzyme has had a product receive the FDA “Breakthrough Therapy” designation and only the second for all of Sanofi! The NNPDF has also been advised that the foundation’s participation, along with our NPB patients and family membership in the April 29th, 2015 ground-breaking meeting with FDA regulatory representatives, played a key role in ensuring that this FDA “Breakthrough Therapy” designation was granted. The opportunity afforded to the NPB (ASMD) patient community through that meeting and this designation is ground-breaking for the Rare Disease Community.

The meeting objective from April 29th, 2015 was described and developed under the following guideline:

Under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), FDA committed to more systematically gather patient’s perspectives of their condition and available therapies to treat their condition. The upcoming meeting on April 29th with the NNPDF and some of their members is an opportunity for FDA and members of the Niemann-Pick community to engage in a unique dialogue to help ensure the patient perspective of living with Niemann-Pick B is better understood. This meeting represents a new opportunity for the rare disease community to continue their partnership with FDA.

CAMBRIDGE, Mass. - Genzyme, a Sanofi company, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olipudase alfa. This enzyme replacement therapy is being investigated for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B, as opposed to type A which is characterized by neurological involvement. ASMD is a serious and life-threatening disorder caused by insufficient activity of the enzyme acid sphingomyelinase (ASM), which results in toxic accumulation of sphingomyelin. There are currently no approved treatment options for patients with Niemann-Pick disease type B.

Breakthrough Therapy designation is intended to expedite the development and review of investigational new drugs that target serious or life-threatening conditions. The criteria for granting Breakthrough Therapy designation are preliminary clinical evidence of substantial improvement on a clinically significant endpoint over available therapies. The Breakthrough Therapy designation is distinct from the FDA’s other mechanisms to expedite drug development and review, and will allow for a close collaboration between Genzyme and the FDA on the olipudase alfa development program.

According to the FDA website the benefits of a breakthrough therapy designation are:

Breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

A breakthrough therapy designation conveys all of the fast track program features (see below for more details on fast track designation), more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review. Section 902 of FDASIA requires the following actions, as appropriate:

  • holding meetings with the sponsor and the review team throughout the development of the drug
  • providing timely advice to, and interactive communication with, the sponsor regarding the development of the drug to ensure that the development program to gather the nonclinical and clinical data necessary for approval is as efficient as practicable
  • taking steps to ensure that the design of the clinical trials is as efficient as practicable, when scientifically appropriate, such as by minimizing the number of patients exposed to a potentially less efficacious treatment
  • assigning a cross-disciplinary project lead for the FDA review team to facilitate an efficient review of the development program and to serve as a scientific liaison between the cross-discipline members of the review team (i.e., clinical, pharmacology-toxicology, chemistry, manufacturing and control, compliance) for coordinated internal interactions and communications with the sponsor through the review division’s Regulatory Health Project Manager
  • involving senior managers and experienced review staff, as appropriate, in a collaborative, cross-disciplinary review

Visit the NNPDF Enzyme Replacement Therapy web page to view the full press release, for more information regarding the FDA's Breakthrough Therapy & see the full history of the ERT clinical trial campaigns!

[Jun 4th, 2015 ~ blg]

 

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MAY/JUNE 2015 CCNNPDF e-Newsletter ~ Now Online

news

Dear Niemann-Pick Disease Families and Friends,

The Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) is excited to introduce our very first, but certainly not our last, monthly e-Newsletter! 

The May/June 2015 e-Newsletter highlights include:

  • Think Again. Think NPC. CANADA!!
  • The 23rd Annual NNPDF Family Support and Medical Conference
  • Research Progress Reports
  • Clinical Trial News and Updates
  • CCNNPDF Membership ~ Action Required 
  • CCNNPDF Fundraising
  • Meet the CCNNPDF Members of the Board

 

Click here to view the CCNNPDF May/June 2015 e-Newsletter!

[June 2nd, 2015 ~ blg]

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Keaton J. Smith
09/24/2000 ~ 05/22/2015

Keaton
Keaton J. Smith (NPC)

Dear NNPDF Families and Friends,

It is with a heavy heart that we notify our NPD family membership of the passing of Keaton J. Smith (September 24, 2000 - May 22, 2015 ~ NPC), who courageously fought the NPC battle for 14 years.  Keaton played on the Tippy Stars Baseball Team and was a huge fan of Purdue men’s and women’s basketball.  He was an 8th grader at Battle Ground Middle School and a member of St. Thomas Aquinas Center in West Lafayette, Indiana. 

Keaton’s family noted that they draw some comfort in knowing that he is now playing with his brother Braden and sister Riley who both also suffered from Niemann-Pick Disease and passed away in 2006 and 2014 respectively.

To Keaton’s loving parents, Trent and Julie, and beloved older sister, Chandlar, please know that your NNPDF family is here for you and to let us know if you need anything in the coming days, weeks and months ahead.  We are holding you all in our hearts and thoughts.

Keaton’s obituary can be found at http://www.soller-baker.com/memsol.cgi?user_id=1588635.

[May 26th, 2015 ~ blg]

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MAY 2015 e-Newsletter ~ Now Online

Banner

Dear NNPDF Families and Friends,

The NNPDF May 2015 e-Newsletter is available! Highlights include:

  • 23rd Annual NNPDF Family Support and Medical Conference
  • Update ~ A Message from your Foundation Board of Directors! 
  • NNPDF Membership ~ Action Required 
  • NNPDF Fellowship Research Progress Reports
  • Clinical Trial News & Updates 
  • Fundraising & Recipes for Success

Click here to view the NNPDF May 2015 e-Newsletter!

[May 19th, 2015 ~ blg]

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Orphazyme Announcement:
Orphazyme Clinical Trial Development Update on Arimoclomol in NPC
Dateline: 05/11/2015

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation has been advised by Orphazyme that an “Observational” clinical study for NP-C, tied to their clinical development program of arimoclomol in NPC, has been registered and made available on www.clinicaltrials.gov.  The link to the information pertaining to just this trial is at: https://clinicaltrials.gov/ct2/show/NCT02435030?term=02435030&rank=1

This information was updated as of April 30, 2015. 

At this time, no site in the United States has been announced and currently the study is not open for participant recruitment.  Orphazyme has indicated that they are targeting a clinical trial to start date in the United States toward the end of first quarter 2016.  As we receive further announcements and updates, it will be posted to our NPD family community! 

You can read either the full text view or the tabular view depending upon your preference.  The text allows you to see the inclusion and exclusion information along with the primary outcome measures they will be looking at. 

Sincerely,

Nadine M. Hill
Executive Director
National Niemann-Pick Disease Foundation

[May 11th, 2015 ~ blg]

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A Re-Introduction to the Canadian Chapter of the National Niemann-Pick Disease Foundation

CCNNPDF

Dear NNPDF Families and Friends,

Many of you may not be aware of the fact that NNPDF has a sister chapter in Canada.  The Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) and the NNPDF work collaboratively to help support families and research to find treatments and a cure for all types of Niemann-Pick Disease.  The NNPDF office in the United States provides administrative, marketing and family support for the CCNNPDF.  Please see the new CCNNPDF e-newsletter to learn more about what is on the horizon for our families and friends in Canada.

Click here to view the CCNNPDF e-Newsletter

All my best,

Nadine Hill
NNPDF Executive Director

[May 7th, 2015 ~ blg]

 

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Vtesse

Vtesse Webinar Summary & SoundCloud Podcast with CEO Ben Machielese

Dear NNPDF Families and Friends,

The NNPDF Central Office is pleased to provide our Niemann-Pick type C family membership community with a summary from the April 16th Vtesse sponsored webinar for those of you who could not be in attendance. Please find the PDF document via this link: http://www.nnpdf.ca/Vtesse.html#Podcast

The Vtesse webinar addressed the progress and updates tied to plans for the VTS-270 (cyclodextrin) Phase II & III clinical trials for Niemann-Pick Disease, Type C (NPC). To see an agenda of the topics discussed during the call ~ please follow this link: April 16th Vtesse sponsored webinar

In addition, we are including a podcast with Ben Machielse, CEO of Vtesse, Inc as he discusses:

  • What is Niemann-Pick Disease type C.
  • The role that NPC parents & patients have played in getting the research moving forward.
  • How NIH & NCATS paved the way for the Cyclodextrin clinical trial.
  • Vtesse, Inc, Cydan and how they became involved with Cyclodextrin.

Visit the Vtesse page for the PDF Webinar summary and Podcast.

[May 5th, 2015 ~ blg]

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FDA & NPB Patient Groundbreaking Meeting
held on
Wednesday, April 29th, 2015


NNPDF & Genzyme FDA Panel Representatives
in front of the Food and Drug Administration Building at
the White Oak Campus in Silver Spring, Maryland.

Front row:  Lillian Saeger (NNPDF), Trina Paulk (NNPDF), Sharon Tan (Genzyme), Sandra Cowie (NNPDF),
Ana Puga (Genzyme) Back row:  Rumana Haque-Ahmed (Genzyme), Joshua Karie (NNPDF), Jamie Ring (Genzyme),
Nadine Hill (NNPDF), Elissa Miller-Visoky (NNPDF), Sherwin Sattarzadeh (Genzyme)

Dear NNPDF Families and Friends,

Recently, the leadership of the NNPDF was approached by representatives from Genzyme (A Sanofi Company) regarding a ground-breaking opportunity for our Niemann-Pick Type B patient community.  Representatives from the United States Food and Drug Administration (FDA) involved with the regulatory aspects of the upcoming Genzyme pediatric and adult Enzyme Replacement Therapy Clinical Trials had asked to meet with members of the NPD Type B patient community.

The meeting objective was described and developed under the following guideline:

Under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V), FDA committed to more systematically gather patient’s perspectives of their condition and available therapies to treat their condition. The upcoming meeting on April 29th with the NNPDF and some of their members is an opportunity for FDA and members of the Niemann-Pick community to engage in a unique dialogue to help ensure the patient perspective of living with Niemann-Pick B is better understood.  This meeting represents a new opportunity for the rare disease community to continue their partnership with FDA.

The NNPDF was thrilled to be able to assist in coordinating this meeting which took place on Wednesday, April 29th at the FDA offices in Silver Spring, Maryland.  The NPB patient representatives from our Niemann-Pick community had the opportunity to share their stories with the FDA.  As Executive Director of the NNPDF, Nadine Hill was honored to be asked to moderate the session between patients and parents of patients living with NP disease, who in turn, engaged reviewers at the Division of Gastroenterology and Inborn Errors Products and senior FDA staff in an open discussion of diagnostic history, symptoms, and challenges associated with their disease.

Approximately fifteen FDA representatives listened attentively and asked clarifying questions probing the variability and heterogeneity of NP disease signs and symptoms.  The meeting had a very positive tone with FDA staffers creating a comfortable and informal environment allowing for a back and forth discussion with the NP representatives.

When asked about their impressions of the meeting, the FDA regulatory project manager and lead medical reviewer noted that these discussions provide tremendous value to the FDA.

Deep gratitude and thanks go out to the NPD Type B patient panel participants without whom this meeting would not have been possible. A special thank you to Alan Gilstrap and Jamie Ring from Genzyme's Patient Advocacy group for their support of this successful meeting.

The NNPDF will provide further updates and feedback from this meeting and the impending US ASMD patient clinical trials as they become available.

Genzyme Attendees:

  • Jamie Ring – Vice President, Patient Advocacy-Rare Diseases and Humanitarian Programs
  • Rumana Haque-Ahmed – Associate Vice President, Regulatory Affairs Rare Diseases
  • Sharon Tan – Global Project Head, Niemann-Pick
  • Ana Puga – Medical Director, Clinical Development Rare Diseases
  • Sherwin Sattarzadeh – Director, Regulatory Affairs Rare Diseases

NNPDF Family Membership Representatives:

  • Sandy Cowie ~ NPB Adult Patient; Toronto, Canada
  • Trina Paulk ~ NPB Adult Patient; Douglasville, Georgia
  • Joshua Karie ~ NPB Adult Patient; Gilbert, Arizona
  • Elissa Miller ~ Parent of a NPB pediatric patient; New York, New York
  • Lillian Saeger ~ Parent of a NPB pediatric patient; Severna Park, Maryland
  • Nadine Hill ~ NNPDF Executive Director; Fort Atkinson, Wisconsin

[May 1st, 2015 ~ blg]

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Genzyme logo

 

Update from Genzyme on
Acid Sphingomyelinase Deficiency (ASMD) Development
Efforts Pediatric Trial
April 24th, 2015

 

Dear NNPDF Families and Friends,

The NNPDF Central Offices have been notified that Genzyme, a Sanofi Company, has released a statement with additional details about the pediatric Phase 1 / 2 clinical trial of recombinant human acid sphingomyelinase (rhASM).

Visit the NNPDF Enzyme Replacement Therapy web page to view the the statement

[Apr 24th, 2015 ~ blg]

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Banner

Dear NNPDF Families and Friends,

The NNPDF April 2015 e-Newsletter is available! Highlights include:

  • 23rd Annual NNPDF Family Support and Medical Conference ~ Dates & Hotel Location
  • Welcome ~ New NNPDF Board Members and Staff
  • NNPDF Fellowships ~ Request for Applications - Deadline: May 15th, 2015
  • Clinical Trial Updates
  • NNPDF Equipment Exchange

Click here to view the NNPDF April 2015 e-Newsletter!

[Apr 21st, 2015 ~ blg]

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Samantha Juliet Emmons
"Sammy"
12/27/2001 ~ 04/16/2015
Sammy

Dear NNPDF Families and Friends,

It is with great sadness that the NNPDF Central office has learned that the 13 year old daughter of our NNPDF family, Brandy and Jeff Emmons has passed away unexpectedly.  Samantha Juliet “Sammy” Emmons was a beloved sister to Alex, Rissa & Liz.  Two of her sisters are courageously fighting the NPC battle. 

Sammy was a 7th grader at South Ogden Junior High, where she excelled in math, art and music “P4SE”. She was an amazing artist, loved playing video games, she was awesome with animals, and loved roasting peeps in the backyard fire pit. 

To sweet Samantha’s mom and dad, Jeff and Brandy please know your NNPDF family is here for you and to let us know if you need support in the coming days, weeks and months ahead.  May all your happy, loving and sweet memories of Sammy help to ease the sadness you are feeling right now and offer you a sense of comfort.  

Click here to view service information and to send condolences

[Apr 20, 2015 ~ blg]

 

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A Message from Jill Flinton, NNPDF Finance Chair
Dateline: April 16th , 2015

Jill Daniel
Jill Flinton 
Mother of...
... Daniel Flinton, NPC  
12/07/06 - 11/11/11

Dear NNPDF Families and Friends,

New for 2015, the NNPDF Board of Directors rolled out a new communication strategy which we hope will assist you in feeling more connected with the foundation and your Board Representatives. This new communication strategy offers us a unique opportunity to share on-going foundation efforts, programs, projects and activities being addressed on your behalf, our family membership and NNPDF key stakeholder’s.

Today, we wish to share with you an update from the 2015 Finance Committee Chair, Jill Flinton, of Charlton, New York, who serves on the foundation board in a volunteer capacity and in memory of her son, Daniel.

Click here to view Jill Flinton's Message to the Community
Dateline: April 16th, 2015

Click here to view Lisa Chavez's Message to the Community
Dateline: March 31st, 2015

Click here to read Board Chair. Leslie Hughes' Message to the Community
Dateline March 24th, 2015

[April 16th, 2015 ~ blg]

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Orphazyme Announcement:
Orphazyme Kicks off Clinical Program of Arimoclomol
in Niemann-Pick Disease Type C
Dateline: 04/15/2015

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation is pleased to share with you the following announcement received from Orphazyme ApS of an upcoming clinical trial for Niemann-Pick Disease Type C.  Orphazyme ApS (Copenhagen, Denmark) develops new therapies for the treatment of rare and genetic diseases.

To read the full press release, visit the NNPDF Orphazyme page


As more information becomes available we will continue to update this page, as well as, our social media sites.

[April 15th, 2015 ~ blg]

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Vtesse

Register for Webinar on Cyclodextrin for NPC
Dateline: April 16th, 2015

Vtesse invites you to hear about their progress, ask questions and provide feedback

 

Dear NNPDF Niemann-Pick Type C (NPC) Family Members,

Many of you have probably been looking forward with great anticipation to the progress updates on Vtesse, Inc.’s plans for VTS-270 (cyclodextrin) for Niemann-Pick Disease, Type C (NPC), announced in January 2015.  

The National Niemann-Pick Disease Foundation (NNPDF) is pleased to be able to share with you an opportunity to attend a town hall webinar hosted by the company’s leadership at Vtesse.  As mentioned in their initial announcement, Vtesse is committed to keeping the community fully informed and soliciting feedback as they move forward with clinical development. The company has informed us it is still in the process of planning the pivotal clinical trial, and as such, it will not have all the answers to your questions. However, this webinar offers an exciting opportunity to be part of the discussion with Vtesse to help shape certain aspects of the company’s plan.  We hope that the audience will consist of patients, their families and clinicians.

A 90-minute webinar will be held in English on April 16, 2015 at 6:30 PM CT geared toward a US and Canadian audience and one geared toward the EU Audience at 6:30 PM GMT.  As a participant, you can expect to:

  • Meet the company’s leadership
  • Hear the company’s view on the advantages of conducting a clinical trial
  • Get a family perspective on participation in Phase I of the Cyclodextrin trial at NIH
  • Hear the company’s preliminary thoughts on the clinical trials
  • Provide feedback to the company on certain aspects of its clinical trial plans
  • Ask any questions that you may have

It is hoped you will take advantage of this opportunity to begin a two-way dialogue and receive information and provide input to the company on the exciting possibilities that lie ahead. Please feel free to reach out to me at nnpdf@nnpdf.org if you have any questions.

For our family membership residing in Europe, Vtesse is hosting a call April 16, 2015 at 6:30 PM BST; 7:30 PM CET aimed at the UK and European Audience. Although the Webinars are aimed at audiences from the USA, Canada and Europe, participants are welcome from all around the world.

  • Register:  US and Canadian Audience, 6:30 PM CDT*, April 16, 2015 ~ http://bit.ly/NPCWebinar
    • *4:30pm Pacific, 5:30pm Mountain, 6:30pm Central, 7:30pm Eastern
  • Register (UK and European Audience), 6:30 PM BST; 7:30 PM CET, April 16, 2015:http://bit.ly/NPC_Webinar   

Sincerely,

Nadine M. Hill

Nadine M. Hill
Executive Director
National Niemann-Pick Disease Foundation

[Apr 7th, 2015 ~ blg]

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A Message from Lisa Chavez, NNPDF Nominations Chair
Dateline: March 31st, 2015

Chavez Breann Chavez
Lisa Chavez 
Mother of...
...Breann Chavez, NPC
2/10/03 - 6/8/06

Dear NNPDF Families and Friends,

Last week the NNPDF Board of Director’s rolled out a new website page designed specifically for Board related updates which will provide a NEW way for your Board Representatives to communicate with you, our family membership and key stakeholder’s.

As we shared with you last week, the NNPDF Board of Director’s wrapped up our annual board meeting earlier this month in Chicago, Illinois. Last week Tuesday, March 24th, 2015, the first post came from the 2015 NNPDF Board Chair, Leslie Hughes, who resides in Wichita Falls, Texas and serves on the foundation board in memory of her great-niece, Riley Corbitt.

Today, we wish to share with you an update from the 2015 Nominations Committee Chair, Lisa Chavez, of Farmington, New Mexico, who serves on the foundation board in memory of her daughter, Breann.

Click here to view Lisa Chavez's Message to the Community
Dateline: March 31st, 2015

Click here to read Board Chair. Leslie Hughe's Message to the Community
Dateline March 24th, 2015

[March 31st, 2015 ~ blg]

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Ashlyn P. Purpura
04/19/2008 ~ 03/25/2015

Ashlyn
Ashlyn (NPC) and her little sister Arieanna

Dear NNPDF Families and Friends,

It is with heavy hearts that the NNPDF Central Office shares word from the home of Kelly and Neil Purpura, that cherished daughter, Ashlyn P. Purpura, lost her battle with Niemann-Pick Disease type C at the tender age of 6 years. 

In addition to her parents, Ashlyn is survived by her little sister, Arieanna.  Ashlyn is described by those who loved her as loving to play and make friends with everyone in her class, she was especially fond of everything pink and purple, and her favorite pastime was listening to music and story time.

Kelly, Ashlyn's mother, shared this over Facebook:

"I want to personally Thank All of you for the condolences they really mean a lot to us.  We are incredibly grateful for all the love and support from the community for our entire family...(A Request from us…..) Everyone is encouraged to wear bright colors on Sunday.  She, Ashlyn, would love to see all the pretty colors. Her favorites are purple and pink but bright if you don't have either. Thanks again"

The family has generously directed that memorial donations in Ashlyn's name be sent to the NNPDF. You can read Ashlyn's service information and send personal condolences to the family by clicking here.

To the Purpura family and all those who held Ashyln dear, please know that we send our deepest and heartfelt sympathies to you and all those whose lives were touched by your sweet  Ashlyn. There are bound to be difficult and trying times ahead, but please know that we, as well as, your extended NPD family community will all be here to give you strength in the coming days, weeks and months ahead.  May the love and support of all those who loved Ashlyn provide you comfort at this time.

[Mar 27th, 2015 ~ blg]

 

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NNPDF Request for Fellowship Applications
2015 Research Fellowships Open

Science

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation invites applications for research fellowships examining the biology of Niemann-Pick Type C (NPC) disease, a lethal neurodegenerative disease for which there are no effective therapies.

Those eligible to apply for funding are:

· Predoctoral students with a lab selected and an approved thesis
· M.D., Ph.D and D.V.M postdoctoral researchers
· Early career investigators

Preference will be given to research projects developing new therapies for NPC, and translational research projects that improve our understanding of the biology, pathogenesis and potential treatment of NPC disease.

For additional information, please visit the NNPDF Research Funding Strategy web page

[March 26th, 2015 ~ blg]

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Tristan Nycole Lindsey
04/09/2002 ~ 03/24/2015

Tristan
Tristan Nycole Lindsey (NPC ~ 12 years)

Dear NNPDF Families and Friends,

The NNPDF Central Office received the heartbreaking news today that precious Tristan Lindsey, beloved daughter of Tracy & Derrick Lindsey, lost her battle with Niemann-Pick Disease type C on March 24th, 2015 at the tender age of 12 years.

In remembrance of her daughter, Tracy lovingly wrote:

"It is with a peaceful heart that I share our daughter Tristan peacefully returned home to our Father in Heaven. She was such a blessing to our family and everyone who knew her. Her beautiful smile and eyes that could say a million words with out her ever speaking, will forever be missed. I know she's running, playing and eating everything in sight. Thank you everyone for loving our baby and loving us as well.

~ Love the Lindsey Family
"

May the family and friends of sweet Tristan find the strength they need to move on in the days, weeks and months ahead. Please know that all the families and friends of the National Niemann-Pick Disease Foundation are with you all in our hearts and in grief. Please know if you should you need us, we are here always with open arms.

Click here for Tristan's service information and to send condolences to the family

[March 25th, 2015]

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Vtesse

~Vtesse, Inc. Press Release~ 
Dateline: 03/25/2015

Vtesse, Inc. Forms Scientific Advisory Board

Dear NNPDF Families and Friends,

We are pleased to share with the foundation's Niemann-Pick Disease type C (NPC) families and community a press release regarding the recently incorporated, Vtesse, a rare disease company spun-off from Cydan Development, Inc.

Vtesse announced today the formation of its Scientific Advisory Board (SAB), to include Niemann-Pick Disease experts Drs. Paul Gissen, Marc Patterson and Forbes D. Porter (who both are currently are on the NNPDF's International SAB), along with biopharmaceutical industry leader Dr. Cristina Csimma, to join Vtesse in their quest to develop new treatments for people facing Niemann-Pick Disease Type C and rare diseases.

Visit the Cyclodextrin page for the full press release

[March 25th, 2015 ~ blg]

 

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A Message from Leslie Hughes, NNPDF Board Chair
Dateline: March 24th, 2015

Leslie Hughes Riley Corbitt
Leslie Hughes 
Great-Aunt of...
...Riley Corbitt, NPC
07/27/02-10/29/08

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation (NNPDF) Board of Directors is pleased to announce a new feature to the NNPDF web site titled, ‘From the Board’. This web page has been created as a way for you to get the latest updates directly from the members of your Board as to the work which is addressed at the foundation and within the various committees. The Board is continuously looking for ways to best communicate information and updates. This is the first of upcoming messages from your ‘Board of Directors’ over the next few weeks and months ahead and we will continue to provide updates periodically as to the“happenings” of the Board and foundation as we move on through the year. 

“The NNPDF Board of Director’s has just completed our annual meeting and wish to provide the entire NPD community with an update. Through this link you will find a new page on the website designed specifically for Board related updates. First up, a message from the 2015 NNPDF Board Chair, Leslie Hughes.”

Please follow this NEW web link below for an update to the NNPDF membership from the Foundation Board Chair, Leslie Hughes.

Click here to view Leslie Hughe's Message to the Community

[March 24th, 2015 ~ blg]


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Banner

Dear NNPDF Families and Friends,

The NNPDF March 2015 e-Newsletter is available! Highlights include:

  • 23rd Annual NNPDF Family Support and Medical Conference ~ Dates & Hotel Location
  • Coach Matt Painter wins $100,000 for NPC Research
  • World Rare Disease Day 2015
  • Vote Jim Green ~ INPDA ~ United Kingdom Volunteer of the Year
  • 2015 NNPDF Annual Board Meeting

Click here to view the NNPDF March 2015 e-Newsletter!

[Mar 17th, 2015 ~ blg]

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Phase 2/3 Adult and Phase 1/2 Pediatric Clinical Trial of rHASM for ASMD
Clinical Trial Update

Dear NNPDF Families and Friends,

Representatives of the National Niemann-Pick Disease Foundation (NNPDF), Niemann-Pick UK and the International Niemann-Pick Disease Alliance (INPDA) today participated in a teleconference with Genzyme, A Sanofi Company.

We were pleased to learn that plans for the Phase 2/3 Adult and Phase 1/2 Pediatric Clinical trial of rhASM for ASMD Niemann-Pick disease continue to progress. Discussions continue with the regulatory authorities, including the FDA and the EMA and we expect Genzyme to release a more detailed community update soon.

Please contact the NNPDF Central Offices should you have any questions at: nnpdf@nnpdf.org

Visit the NNPDF Genzyme Clinical Trial page for the history and archive of previous updates on these trials: http://www.nnpdf.org/npresearch_11.html

[Mar 11th, 2015 ~ blg]

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Coach Matt Painter Competes for Local Charity
WLFI.com News Article

Banner

Dear NNPDF Families and Friends,

The Smith family sent us the below video highlighting the efforts of Coach Matt Painter from Purdue University and the Smith Family BReaK-Thru Fund in their quest to win $100,000 for NPC research!

We're 10 days away now and the overall efforts of our collective NPD community has been absolutely amazing! 

Remember to vote daily and share within your own personal circles.  Let's keep this momentum going and see the Smith Family BReaK-thru Fund & Coach Matt Painter to the Championship position!

Vote Today ~ Vote Everyday!

 

[Mar 5th, 2015 ~ blg]

 

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Infiniti Coaches Charity Challenge ~ 2015
$100,000 for NPC Research ~ Just 11 Clicks Away
FINAL ROUND

R4

Dear NNPDF Families and Friends,

The entire Niemann-Pick Disease type C community has been offered an unparalleled opportunity to raise $100,000 towards NPC research, but it will only happen if YOU take action and encourage others to do the same. It's easy! Vote everyday online between now and March 15th to push the Trent and Julie Smith Family Foundation "BReaK Thru Fund" for NPC research to the finish line and the top prize of $100,000! (Three of the Smith family children have been diagnosed with Niemann-Pick Disease type C ~ Braden, Riley and Keaton ~ thus BReaK-Thru Fund.)

Matt Painter, Purdue University Coach and the Smith Family BReaK Thru Fund are working together with the NPC community to vote Matt Painter into the final round! With your help, NPC research WILL garner much needed funds for NPC research, but only if you vote daily! We CAN do this if everyone gets behind this project. 

The FINAL round of voting opened on March 2nd, 2015 and continues through March 15th when one Coach out of the final four will claim the $100,000 grand prize!

Coach Painter is currently neck to neck with another Coach and we want to make sure he is announced as Champion on March 15th!

www.votemattpainter.com

Vote daily, share this with your families, share this with your friends and encourage them to do the same.

Alone we are rare, but together we are strong!

[Mar 4th, 2015 ~ blg]

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WSJ

~Cyclodextrin In the News~

“Deaf or Death?  In Drug Trial, Parents Weigh Life vs. Hearing Loss”
& "Others Hit Unexpected Obstacles in NPC Survival Fight"
By Amy Dockser Marcus ~ Staff writer for the Wall Street Journal
Dateline: March 1st, 2015 
---
"Our Bid to Raise Awareness of Our Son's Disease"
BBC Health News
Dateline: February 27th, 2015

 

BBC

Dear NNPDF Families and Friends,

The NNPDF Central Office was recently notified of three articles "In the News", which address ongoing therapies and family efforts to care for their children for Niemann-Pick Disease type C. The first two articles are highlighted in the Wall Street Journal, the third in the United Kingdom via the BBC. We have included links for you below.

"Deaf or Death?  In Drug Trial, Parents Weigh Life vs. Hearing Loss"
& "Others Hit Unexpected Obstacles in NPC Survival Fight"
Wall Street Journal
Dateline: March 1st, 2015

WSJ

Author, Amy Marcus, of the Wall Street Journal, continues to update her previously published articles on Niemann-Pick Type C, which has focussed on several families journeys to the current Cyclodextrin trials at the National Institutes of Health (NIH). 

The focus of the first article addresses the issue of higher doses of Cyclodextrin and the effects on the patients hearing loss, a side affect that is being weighed against the efficacy of the drug itself.  This is also related to the Press Release from Dr. Porter released on February 27th

The second article follows up with the Papier and Hempel families since their last published articles and the effects NPC has had on their life through their trials with Cyclodextrin.

Click here to read "Deaf or Death? In Drug Trial, Parents Weigh Life vs. Hearing Loss"

Click here to read "Others Hit Unexpected Obstacles in NPC Survival Fight"

 

~~~~

"Our Bid to Raise Awareness of Our Son's Disease"
BBC Health News
Dateline: February 27th, 2015

BBC

This BBC post highlights Sam Evans, NPC, son of Miriam Evans (Administrative Assistant of the International Niemann-Pick Disease Alliance (INPDA)) in a bid to raise awareness for Niemann-Pick disease type C (NPC), as well as, Rare Disease Day on February 28th.

The article also shines light on the INPDA's campaign ~ Think Again. Think NP-C ~ which aims to reduce diagnosis time by helping doctor's unfamiliar with NPC to recognize the key signs and symptoms.

Click here to read the BBC Health News Article

[Mar 2nd, 2015 ~ blg]

 

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Niemann-Pick Disease Clinical Trial Updates:

- Cyclodextrin (NPC ~ pediatric)

- Vorinostat (NPC ~ adult)

- Enzyme Replacement Therapy (ASMD ~ pediatric)

 

Niemann-Pick Disease Research “In the News”:

- Penn Vet Researchers Identify Effective Treatment for Niemann-Pick Disease Type C

Denny Porter

Dr. Forbes D. Porter, MD, PhD
Senior Investigator, PDEGEN, NICHD
Program Head, PDEGEN, NICHD
Clinical Director, NICHD

Dear NNPDF Families and Friends,

In tandem with World Rare Disease Day 2015 and in support of our NPD families WORLD-WIDE, the National Niemann-Pick Disease Foundation (NNPDF) is pleased to share the most recent research and clinical trial updates within the Niemann-Pick Disease Community.

The NNPDF central offices received the following clinical trial updates from Dr. Forbes D. Porter at the National Institutes of Health (NIH) to share with the NPC membership with regards to recent developments in the Cyclodextrin and Histone Deacetylase Inhibitors (HDACi ~ vorinostat) clinical trials.

Click here to read the latest press release from National Institutes of Health (NIH);dtd: February 27th 2015

For a detailed history of leading up to these two clinical trials, you can visit the NNPDF Cyclodextrin web page and the HDACi webpage

1st Contact
Lee Ann Keener

leeann.keener@nih.gov

(301) 594-2005
Clinical Research Nurse
2nd Contact
Nicole Farhat nicole.farhat@nih.gov
(301) 594-1765
Registered Nurse Clinician/Researcher
  Dr. Forbes D. Porter     Prinicpal Investigator

 


Genzyme logo

Genzyme Pharmaceuticals, a division of Sanofi, which most recently announced two upcoming clinical trials to begin in 2015 for our Niemann-Pick Disease Type B adult and pediatric patient community (see the press releases here) developed activities around NPB patient families in support of World Rare Disease Day.   Genzyme employees in the Boston area spent the day Friday, February 27th recognizing their focused and directed efforts of support and research towards patients in the Rare Disease Community.  As the ASMD Enzyme Replacement Therapy pediatric clinical trial will soon begin enrolling, and to help Genzyme employees all over the Boston area understand the impact of Niemann-Pick B disease on an individual’s quality of life, Genzyme hosted two families raising children who are growing up with NPB.  Genzyme hosted events throughout the day at all 6 of their Boston area facilities.  You can receive up-to-the minute updates, as well as, a full recap on the day by following Genzyme on Twitter at @genzymecorp via the handle  #genzymerelay!

Genzy
Kalia & Jack


Niemann-Pick Disease Research “In the News”:

Vite
Dr. Charles Vite, D.V.M., Ph.D.

In conjunction with the clinical trial updates noted above, the NNPDF also received notification of a recent on-line publication highlighting the work of Charles Vite, Ph.D, and the invaluable work that he has been doing with the naturally-occurring Niemann-Pick Type C felines at the University of Pennsylvania ~ School of Veterinary Medicine.

Dr. Vite's work has been co-sponsored by the National Niemann-Pick Disease Foundation (NNPDF) and the Canadian Chapter of the NNPDF (CCNNPDF) since August of 2011. In the summer of 2014, Dr. Vite was granted an extension to his research so as to continue on with his significant work associated with Niemann-Pick Disease Type C. 

An article was published in PennNews online that we wanted to share with the NPC community.

Click here to view the news article

Follow this link to learn more about Dr. Vite's specific research:  http://nnpdf.org/npresearch_05.html

 

[Feb 27th, 2015 ~ blg]

 

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23rd Annual NNPDF Family Support and Medical Conference
"The Winds of Hope are on the Horizon...
... for our NPD Community

Chicago

In support of World Rare Disease Day and our Niemann-Pick Disease Community ~ the NNPDF is pleased to announce the site location of our 23rd Annual Family Support and Medical Conference to be held in Chicago, Illinois in late July, early August!

Plan Your Visit to the Windy City!

As Winter turns to Spring the NNPDF begins to plan our annual NNPDF Annual Family Support and Medical Conference and want to provide our NPD community with some planning tips and guides to help make your trip to the Windy City a GREAT family get-away!  The conference weekend always provides an evening for a family free night, where families can explore the destination city and take in the sights!  The conference hotel destination site is being finalized and we will announce the weekend dates as soon as all is confirmed!  

Chicago has a little something for everyone.  See the links below to start 
planning your trip!

The Navy Pier - You can't truly experience Chicago without a trip to the Navy Pier.  This 50 acre playground of entertainment, museums, activities, restaurants, and shops is the perfect place for some family fun time.

Chicago Museums - From the bottom of the sea to the top of the stars, Chicago has it all on display.  Start your adventures on the famous Museum Campus along Lake Michigan.  Three world-renounced museums call it home: The Adler Plantarium & Astronomy MuseumThe Field Museum, and the Shedd Aquarium.

Chicago Shopping - Introducing the Magnificent Mile: This cosmopolitan, history-packed section of Michigan Avenue teems with shopping, dining and entertainment treasures to make any vacation unforgettable.. 

Chicago Dining - Celebrity chefs? Check.  World-renowned restaurants? Check. Ethnic dining from around the globe? Check.  Chicago's dining scene is unparalleled. 

Sports and Recreation - With passionate fans, star players and the historic Wrigley Field, it is no surprise that Sporting News named Chicago the "Best Sport City".  Whether you enjoy golfing, soccer or baseball, Chicago holds something for every sports fan.


As we get closer, more information will be released via e-Newsletter, the NNPDF website and social medias.  So keep your eyes on your e-mail, this NNPDF Family Conference page, as well as, the Newsline for the latest updates.

[February 27th, 2015 ~ blg]

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Be Creative on World Rare Disease Day 2015
February 28th, 2015 ~ WORLDWIDE!

Rare

Dear NNPDF Families and Friends,

Here is a creative way to help raise awareness for Rare Disease Day in honor your rare NPD loved ones!  If you decide to participate, we would love for you to share it with the NNPDF Staff Facebook page, on the NPD Awareness Facebook page or on Twitter with the hashtag #NNPDF

Be creative and join the NNPDF and NORD for WORLD Rare Disease Day 2015!

[Feb 26th, 2015 ~ blg]

 

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Remind

Dear NNPDF Families and Friends,

Just a reminder that the National Institutes of Health will be having their Twitter chat today with leading experts in the field, including NIH Director Dr. Francis Collins and NCATS Medical Consultant, Dr. John Ferguson. 

Check below for your local time:

2:30-3:30 pm (EST) 
1:30-2:30pm (CST)
12:30-1:30pm (MST) 
11:30am-12:30pm (PST)

You can sign up for a Twitter account at www.twitter.com if you don't already have one. To join the discussion, you'll need to post a question to your Twitter page, using the hashtag #NIHchat with it.  This will alert the hosts of this NIH chat to view your question and they will tag you back in their response.

Here is the NIH page showing their past Q&A sessions to give you a better idea of how they do it: http://www.nimh.nih.gov/health/twitter-chats/index.shtml

If anyone has any questions regarding the Twitter chat today, feel free to contact the NNPDF Offices here on Facebook, by phone (920-563-0930) or via e-mail: nnpdfoffice@idcnet.com

[Feb 25th, 2015 ~ blg]

 

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Dear NNPDF Families and Friends,

Join the National Institutes of Health (NIH) on February 25, 2015 from 2:30-3:30 pm (EST) (1:30-2:30pm (CST), 12:30-1:30pm (MST) & 11:30am-12:30pm (PST)) on Twitter to raise awareness for rare diseases and talk to leading experts in the field, including NIH Director Dr. Francis Collins and NCATS Medical Consultant, Dr. John Ferguson. You must have an active Twitter account to participate in this hour long Q&A with the leaders of the NIH.

To join the conversation, log in to your Twitter account, at the above date and time, and use #NIHchat along with your question to receive a reply.

[Feb 23rd, 2015 ~ blg]

 

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Banner

Dear NNPDF Families and Friends,

The NNPDF February 2015 e-Newsletter is available! Highlights include:

  • Rare Disease Day ~ 2015
  • WORLD Symposium 2015
  • Expanded Access of Compassionate Use
  • Webinar ~ Young Adult Patients Transition to Medicare
  • $100,000 Online Challenge for NPC Research ~ Round 3
  • 2015 NNPDF Annual Board Meeting

Click here to view the NNPDF February 2015 e-Newsletter!

[Feb 17th, 2015 ~ blg]

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NPC (Niemann-Pick Type C) Updates from the 2015 WORLD Symposium

Vite

Dear NNPDF Families and Friends,

Members of the National Niemann-Pick Disease Foundation (NNPDF), Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF), International Niemann-Pick Disease Alliance (INPDA) and the International NNPDF Scientific Advisory Board are currently in attendance at the WORLD Symposium in Orlando, Florida ~ Tuesday, Feb 10th ~ Thursday, Feb 12th, 2015.  The attendees are filling a variety of roles tied to our patient advocacy membership representation, presentations of research and keynote speakers for symposiums addressing the next generation of lysosomal disorders:  Treatment, Biomarkers and Talent needed to succeed in the Future.

Today we're highlighting all the NPC research presented at the WORLD Symposium last week! 

Included is research updates from Dr. Charles Vite. Dr. Vite's work has been co-sponsored by the National Niemann-Pick Disease Foundation (NNPDF) and the Canadian Chapter of the NNPDF since August of 2011.  He was recently granted a second research grant to continue on with this significant research associated with Niemann-Pick Disease Type C. 

To learn more about Dr. Vite's current and previous research history with the NNPDF, visit the NNPDF Sponsored Research Grants web page.

i Intracisternal cyclodextrin ameliorates neurological dysfunction, increases survival time, and stops Purkinje cell death in feline Niemann–Pick type C1 disease

Principal Investigators: Charles H. Vite, Jessica H. Bagea, Gary P. Swain, Maria Prociuk, Tracey U. Sikora, Patricia O'Donnell, Therese Ruane, Sarah Ward, Alexandra Crooks, Su Li, Elizabeth Mauldin, Veronika Stein, Daniel S. Ory, Mark L. Kao, Cristin Davidson, Marie T. Vanier, Steven U. Walkely, University of Pennsylvania, Philadelphia, PA, USA, Washington University School of Medicine, St Louis, MO, USA, Janssen Pharmaceutical Companies of Johnson and Johnson, Titusville, NJ, USA, Albert Einstein College of Medicine, Bronx, NY, USA, Université Claude Bernard, Lyon, France

Click here to read the abstract

i Rapid kinetics of β-cyclodextrin entering and exiting cells: Implication of its mechanism on reduction of
cholesterol accumulation in Niemann
Pick disease type C cells

Principal Investigators: Sheng Dai, Andrés E. Dulcey, Manju Swaroop, Juan Marugan, John McKew, Wei Zheng, National Institutes of Health, Bethesda, MD, USA

Click here to read the abstract

i Improved diagnostics of NiemannPick disease type C by the analysis of plasma oxysterols

Principal Investigators: Janine Reunert, Frank Kannenberg, Manfred Fobker, Thorsten Marquardt, University Hospital Muenster, Muenster, Germany

Click here to read the abstract

i Experience with 7-ketocholesterol and ccl18/parc as surrogated biomarkers in a series of Spanish NiemannPick disease type C patients

Principal Investigators: Jorge J. Cebollaa, Isabel De Castro-Orósc, Pilar Irúna, Pilar Alfonso, Laura López de Frutos, Marcio Andrade-Campos, Miguel Pocovíc, Pilar Giraldo, Universitary Hospital Miguel Servet, Zaragoza, Spain, Aragon Health Sciences Institute (I+CS), Zaragoza, Spain, University of Zaragoza, Zaragoza, Spain, Spanish Foundation of Gaucher Disease and other Lysosomal Disorders (FEETEG), Zaragoza, Spain, Biomedical Network Research Center on Rare Biology and Diseases (CIBERER), ISCIII, Zaragoza, Spain

Click here to read the abstract

i A new formulation for the treatment of neurological and systemic defects in NiemannPick
disease type C

Principle Investigators: Suhail Alam, Michelle Getz, Yana Fedotova, Kasturi Haldar, University of Notre Dame, Notre Dame, IN, USA

Click here to read the abstract

Dr. Edward Schuchman, esteemed member of the NNPDF International Scientific Advisory Board, also presented the below research.

i Novel use of the lysosomal enzyme acid ceramidase for the treatment of inflammatory lung diseases, including cystic fibrosis

Prinipal Investigator: Edward H. Schuchman, John Clancy, Emily Dimango, Irina Petrache, Erich Gulbins, Xingxuan Hea, Icahn School of Medicine at Mount Sinai, New York, NY, SA, Cincinnati Children's Hospital, Cincinnati, OH, USA, Columbia University School of Medicine, New York, NY, USA, Indiana University School of Medicine, Indianapolis, IN, USA, University of Essen, Essen, Germany

Click here to read the abstract

To see all the Niemann-Pick related research presented at the conference, visit the NNPDF WORLD Symposium web page.

[Feb 17th, 2015 ~ blg]

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ASMD (Niemann-Pick Disease Type A/B & B) Updates from the 2015 WORLD Symposium

Wass

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation is excited to share with our NPD community family membership research updates presented at the 2015 WORLD Symposium on Lysosomal Disease Research under the direction of:  Dr. Melissa Wasserstein M.D. & Genzyme, a Sanofi Company.  This research is in support of Genzyme’s ongoing efforts to develop a potential therapy for acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease Types A and B).

Dr. Melissa Wasserstein, a current two-year grant recipient of the NNPDF, in support of her natural history study of NPD type A & B, is attending the WORLD Symposium as a presenter of her current work titled:

"An open-label, multicenter, ascending-repeat-dose study of the tolerability and safety of recombinant human acid sphingomyelinase (rhASM) in patients with ASM deficiency (ASMD)"

We're also excited to share an additional research abstract presented by Dr. Beth Thurberg, MD, PhD, vice president of Pathology at Genzyme Corporation, titled:

"Hepatic pathology of acid sphingomyelinase deficiency: Clearance of sphingomyelin with recombinant human acid sphingomyelinase administration is associated with improvement in pro-atherogenic lipid profiles"

Further, Dr. Thurberg discusses the results of the ERT Phase 1B clinical trial in the video below addressing the clearance of sphingomyelin with recombinant human acid sphingomyelinase administration in patients with Niemann-Pick Type B disease.

The NNPDF Central Offices have been notified that Genzyme, a Sanofi Company, has released an official press announcement with details of the adult Phase 2/3 of recombinant human acid sphingomyelinase (rhASM).

Genzyme Presents Data from its Phase 1b Program for Niemann-Pick Type B at the Lysosomal Disease Network’s WORLD Symposium 2015

Dateline: February 12th, 2015

"CAMBRIDGE, Mass. --Genzyme, a Sanofi company, today presented data from its Phase 1b clinical study at the Lysosomal Disease Network’s WORLD Symposium 2015 in Orlando, Fla. detailing the investigational use of enzyme replacement therapy in the non-neurological manifestations of acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease type B), a lysosomal storage disease caused by genetic mutations that affect the metabolism of sphingomyelin. The Genzyme study evaluated the tolerability and safety of olipudase alfa (recombinant human acid sphingomyelinase) in five adult patients with ASMD. 

Melissa P. Wasserstein, MD, Director of the Program for Inherited Metabolic Diseases; Medical Director of the International Center for Types A and B Niemann Pick Disease, Mount Sinai School of Medicine, presented: An open-label, multicenter, ascending-repeat-dose study of the tolerability and safety of recombinant human acid sphingomyelinase (rhASM) in patients with ASM deficiency (ASMD). In the trial, each patient received a starting dose of intravenous olipudase alfa at 0.1 mg/kg and escalated dosing every two weeks according to a predetermined schedule up to 3 mg/kg or their maximum tolerated dose. The secondary objective was to study the pharmacokinetics, pharmacodynamics, and exploratory efficacy of olipudase alfa administered every two weeks for 26 weeks. The study findings showed that the dose escalation regimen was well tolerated, with all patients reaching the maximum dose of 3 mg/kg. No serious or severe adverse events or deaths were reported. The data presented on the repeat-dose safety, pharmacodynamics, and exploratory efficacy of olipudase alfa support its continued development for the investigational use in non-neurological manifestations of ASMD. All five patients are participating in the Long-Term Study and will continue on therapy.  

"Though a small number of patients, the response we have observed to date is an early indication that this ASM enzyme replacement therapy is promising for this therapeutic area,” said Genzyme’s Acting Head of Rare Diseases, Richard Peters, M.D., Ph.D. “We look forward to continuing this program and learning more as we work toward advancing a treatment option to patients that is both safe and well tolerated.” Genzyme plans to begin enrolling patients in a Phase 2 program for Niemann-Pick Type B in 2015."

Click here to view the official press release in PDF format

The NNPDF and all of the members of our ASMD family community wish to extend a HUGE note of thanks to the 5 Phase 1B clinical trial participants in and taking on this brave effort which involved extreme commitment of time, travel, work and family involvement in support of the wider ASMD (NPD Type B) community and patients worldwide.

We will continue to bring you the latest research from the Symposium floor as it is made available to us.  In the meantime, you can read all the abstract research on the NNPDF WORLD Symposium web page.

**Abstract publication notice.

Published in the February 2015 special “Lysosomes Issue” of Molecular Genetics and Metabolism (MGM). Articles and full text of the abstracts from this issue can be purchased individually from Elsevier. The journal has been published and is available online (click here)

http://www.journals.elsevier.com/molecular-genetics-and-metabolism/open-access-articles/



[Feb 12th, 2015 ~ blg]

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WORLDSymposium 2015

Dear NNPDF Families and Friends,

Members of the NNPDF, CCNNPDF, INPDA and the International NNPDF Scientific Advisory Board are currently in attendance at the WORLD (We’re Organizing Research for Lysosomal Diseases) in Orlando, Florida ~ Tuesday, Feb 10th ~ Thursday, Feb 12th, 2015.  The attendees are filling a variety of roles tied to our patient advocacy membership representation, presentations of research and keynote speakers for symposiums addressing the next generation of lysosomal disorders:  Treatment, Biomarkers and Talent needed to succeed in the Future.

The goal of the WORLD (We're Organizing Research for Lysosomal Diseases) symposium is to provide a forum to discuss the challenges to research and development of treatments for patients with rare diseases, and to identify opportunities to support the advancement of therapeutic options. Clinicians and researchers who work with lysosomal diseases will have the opportunity to learn about the progression of research and therapy approval processes for a variety of different diagnoses and therapies via face-to-face lectures and in-depth discussion with a panel of experts. This symposium is designed to help patient advocates, scientists, clinicians and other health care professionals identify what resources and actions will be needed to move lysosomal disease research forward.

To view those in attendance, as well as, the latest updates and abstracts regarding NPC & ASMD, visit the NNPDF WORLD Symposium web page.

[Feb 11th, 2014 ~ blg]

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Banner

Important Message from the NNPDF Board of Director's

MEMO: To the Membership of the NNPDF.

  FROM: NNPDF Board of Directors (http://www.nnpdf.org/aboutus_03.html)

  DATE: Monday, February 9th, 2015

Dear NNPDF Families and Friends,

It has come to our attention that a recent private Facebook group has been developed containing posts indicating their perspective on the financial status of the National Niemann-Pick Disease Foundation (NNPDF).

On August 28, 2014, members of the NNPDF Board of Directors, along with the foundation’s legal counsel, provided a conference call to all members of the NNPDF.  An announcement pertaining to this informational toll-free NNPDF membership conference call was shared with our foundation membership through various avenues of posts and social media updates.

(*Please see the specific web links below.)

As advised by the foundations legal counsel and, as agreed to by the NNPDF Board of Directors, we responded to a number of accusations regarding the financial status of the NNPDF.  During this call, which was recorded, NNPDF members were afforded the opportunity to submit questions which were addressed during the call.  In addition, an opportunity for follow-up questions was also made available to our foundation members. 

During the conference call we advised the NNPDF membership that an unannounced “special review” of the financial records of the foundation was performed at the NNPDF Central Offices in April of 2014.   The official report from the hired CPA recapping their findings stated that no financial discrepancies were found. 

The unannounced “special review” of the financial records of the foundation did result in several management recommendations, which is standard in any audit report.  Most of these recommendations were already in practice at the NNPDF, and the ones which were not, were evaluated by the full Board of Director’s and were either implemented or found not practical due to the size of our Foundation.

In addition, the Foundation also underwent its own annual audit by a Certified Public Accounting (CPA) firm, performed on August 27th, 2014, which also resulted with no significant findings.  The resulting 2013 National Niemann-Pick Disease Foundation IRS Federal Tax Form 990 may be found on the NNPDF web site at: http://www.nnpdf.org/aboutus_11.html

As the NNPDF Board of Directors indicated during the NNPDF member conference call ~ going forward the NNPDF Board of Directors and Staff would be focusing on our day-to-day operations and providing the essential family program services, informational resources & referrals, and research advances to our families and foundation membership.

To continue addressing these unfounded allegations only serves to take away precious time from the work that the Foundation is committed to providing to our family membership.

We WILL Persevere in OUR Quest for a Cure!

Sincerely,

Your NNPDF Board of Directors  (http://www.nnpdf.org/aboutus_03.html )


*NNPDF Web links as mentioned in the above statement and available to all NNPDF membership:

  [Feb 9th, 2015 ~ blg]

 

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WRDD

Dear NNPDF Friends and Families,

In preparation for World Rare Disease Day, the NNPDF central offices are offering to create custom Facebook & NEW Twitter graphics to honor your NPD loved ones!

Similar to graphics done in the past, we've created these for our families to help spread awareness between now & World Rare Disease Day on February 28th, 2015.  This year we've expanded to Twitter covers for those who are more inclined to use that social network.

To request a custom cover/icon:

  1. E-mail the NNPDF Central Office at: bgreen@nnpdf.org.
  2. Include a favorite photo of your child (.jpeg or .png is preferred) and advise of their name (first and last), and disease type (NPC, NPA, NPB).  If you have sent us a photo previously for a custom  graphic, you can specify to use the last photo you sent as well.
  3. Specify if you are in the US, Canada or UK for the appropriate banner
  4. You can request graphics for Facebook, Twitter or both if you prefer.

We will be happy to quickly create the following graphics for you to use. The NNPDF Central Office will e-mail these directly back to you.

Spencer sp
T M
e s

~ E-mail: bgreen@nnpdf.org with the requested information above.  The NNPDF will develop and e-mail back to you in time for Rare Disease Day 2015! 

Keep an eye on the NNPDF World Rare Disease web page for additional updates as we get closer to February 28th, 2015.

[Feb 4th, 2014 ~ blg]

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lNPC

2014 Million Dollar Bike Ride Funds
UPDATE

Dear NNPDF Friends and Families,

The NNPDF Central Offices are pleased to share with our NPC Community how the funds raised by Team NPC and the collective efforts of the Reedy-DiGiovani, Mayo, Coppola & Honsinger families have been applied!

We received word from University of Pennsylvania that the funds are to be allocated towards research as noted below:

The NPC grants were awarded to:

Pilot grant for $50,000

Ashley Bush, MB BS, DPM, FRANZCP, PhD, FTSE
NHMRC Australia Fellow
The Florey Institute of Neuroscience and Mental Health

“Dual targeting of defective lipid and metal pathways in Niemann-Pick type C disease: an in vitro pilot study”

 
Postdoctoral fellowship for $35,000

Brittney Gurda, Ph.D.

University of Pennsylvania, School of Veterinary Medicine

“CNS gene therapy to restore NPC1 protein expression in cerebellar Purkinje cells and delay or prevent disease onset in feline Niemann-Pick type C1 (NPC1) disease”

Team NPC ~ your efforts made this possible and we want to thank you again for all the work you do to further NPC research!  You are an inspiration on how to Persevere in our continued, collaborative Quest for a Cure!

GO TEAM NPC!!!

Team NPC

[Jan 29th, 2015 ~ blg]

 

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Infiniti Coaches Charity Challenge ~ 2015
Round 2 ~ January 26th - February 15th, 2015

Round2

Dear NNPDF Families and Friends,

As of January 26th through February 15th, 2015~ Round 2 of the Infiniti Coaches' Charity Challenge is underway! Matt Painter, Purdue University coach, is representing the Smith Family BReaKthru Fund to win upto $100,000 for NPC research!

Currently, Coach Painter is tied at 24% with another participant, so let's keep the momentum going for this round and into the remaining rounds.  Remember to vote daily for Coach Painter and let's help get him to the $100,000 Champions spot this year!

Click Here to Vote for Matt Painter

For additional information and rules on the challenge: Click Here

[Jan 29th, 2015 ~ blg]

 

Genzyme logo

 

Update from Genzyme on
Acid Sphingomyelinase Deficiency (ASMD) Development Efforts Adult Trial
January 28th, 2015

 

Dear NNPDF Families and Friends,

The NNPDF is pleased to provide you with the following update from Genzyme, a Sanofi Company, on NEWLY released information about the adult Phase 2 / 3 clinical trial of recombinant human acid sphingomyelinase (rhASM).

Visit the NNPDF Genzyme web page to view the full announcement

Please be advised of the following details associated with the adult Phase 2 / 3 clinical trial #:  NCT02004691:

  • The study is NOT yet open for patient recruitment
  • Inclusion / Exclusion criteria are provided in the post
  • Estimated enrollment of 35 adult patients
  • MUST be 18 years and older
  • The number of country clinical sites and locations has NOT yet been determined ~ but there will be clinic sites in multiple countries.
  • More information is detailed at the trial page link here.  

[Jan 28th, 2015 ~ blg]

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FDA Grants Orphan Drug Designation for Arimoclomol
Dateline: 01/27/2015

Dear NNPDF Families and Friends,

Orphazyme today announced that the Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted an orphan drug designation for Orphazyme’s Arimoclomol as a new treatment for Niemann-Pick disease type C.

The US orphan drug designation complements the corresponding EMA orphan drug designation that was granted for the program in 2014.

The recent successful completion of a €20m Series B financing round, together with the FDA and EMA orphan drug designations, establish a solid foundation for initiating our clinical studies in NP‑C with the purpose of providing arimoclomol as a safe and effective therapeutic option to patients suffering from this devastating disease as well as relief to their families,” says Dr. Carlos R. Camozzi, Chief Medical Officer of Orphazyme.

Click here to view the full press release

“The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions(http://www.nnpdf.org/CyberCautions.html) about medical information provided on the Internet."

[Jan 27th, 2015 ~ blg]

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Parker Maddox Stults
January 8th, 2006 ~ January 23rd, 2015

Parker

Dearest NNPDF Families and Friends,

It is with profound heartbreak that we advise our NPD membership community that Parker Maddox Stults, one of our brave and courageous Niemann-Pick type C children, lost his fight against NPC on January 23rd, 2015 at the tender age of 8 years.  Parker is survived by his parents, Jennifer & Michael Stults, & by his younger brother Duncan. He was proceeded in death by his older brother Brisan less than a year ago, who passed on March 4th, 2014 from NPC at the age of 9 years.

Parker's mother, Jennifer, wrote of her son on Facebook:

"Parker flew into the arms of Jesus and was welcomed into Heaven by his big bubbas Brisan. Many that loved him were here to comfort him and love on Michael, Duncan and myself. Our two precious boys are together once again."

Visitation will be held Thursday, January 29th, 2015 from 5:30pm - 7:30pm at the Pleasant Valley Baptist Church in Liberty Missouri.  Funeral services will be held Friday, January 30th, 2015 at 10:00am. 

Our thoughts and sympathies are with little Parker's family during this time of heartbreak and loss.   Please know that your NPD family and community grieves with you and will be keeping all who loved Parker in our hearts in the days, weeks and months ahead.  

Should you need us, know we are here with open arms ~

[Jan 26th, 2015 ~ blg]

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Infiniti Coaches Charity Challenge ~ 2015
January 5th ~ March 15th, 2015

CCC

Dear NNPDF Families and Friends,

The entire Niemann-Pick Disease type C  community has been offered an unparalleled opportunity to raise $100,000 towards NPC research, but it will only happen if YOU take action and encourage others to do the same. It's easy! Vote once a day online between now and March 15th to push the Trent and Julie Smith Family Foundation "BReaK Thru Fund" for NPC research to the finish line and the top prize of $100,000! (Three of the Smith family children have been diagnosed with Niemann-Pick Disease type C ~ Braden, Riley and Keaton ~ thus BReaK-Thru Fund.)

Click Here to Vote for Matt Painter!

This on-line voting contest is sponsored by, The Infiniti Coaches Charity Challenge, in for ten (10) weeks, the nation’s top coaches will be counting on fans like you to vote for their favorite charity. Vote for your coach’s favorite charity. Help increase their chances by sharing with your friends. The coaches receiving the most votes gain a spot in the finals, with the winning coach receiving $100,000 toward his charity (each coach will get at least $1,000 for his favorite cause). So start voting now!


Matt Painter, Purdue University Coach and the Smith Family BReaK Thru Fund are working together with the NPC community to vote Matt Painter into the final round! With your help, NPC research WILL garner $100,000, but only if you vote daily! We CAN do this if everyone gets behind this project. 

The first round of voting opened on January 5th, 2015 and continues through January 25th, 2015 when 24 Coaches out of the initial 48 will advance to round two of four total.  Vote daily for Matt Painter to ensure he makes it to the final round!

Share this with your families, share this with your friends and encourage them to do the same.

Alone we are rare, but together we are strong!

[Jan 22nd, 2015 ~ blg]

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A Sincere Thank You
From the APMRF, DART, SOAR & NNPDF!

Dear NNPDF Families and Friends,


We would like to share this thank you letter, written collaboratively by the Ara Parseghian Medical Research Foundation (APMRF), Dana's Angels Research Trust (DART), Support of Accelerated Research for NPC Disease (SOAR) & the National Niemann-Pick Disease Foundation (NNPDF), with regards to the earlier press release from Vtesse, Inc. 


That announcement dated, January, 7th 2015, highlighted the transition of the National Institutes of Health (NIH) / National Center for Advancing Translational Sciences (NCATS) Cyclodextrin clinical trials from a public project to a public/private partnership with Vtesse, Inc.  

Thank


    Ara Parseghian 
Medical Research Foundation~APMRF
www.parseghian.org

      Dana’s Angels Research Trust
~DART

www.danasangels.org

      Support Of Accelerated Research for NPC Disease ~ SOAR www.hideandseek.org

     National Niemann-Pick 
Disease Foundation 
~NNPDF

www.nnpdf.org

Vtesse, Inc. 
www.vtessepharma.com

      January 7th, 2015 ~ 
Vtesse Press Release and Announcements:  

www.nnpdf.org/Cyclodextrin.html

The NNPDF Central Offices will continue to assist with the dissemination of information pertaining to the news and updates relating to the ongoing efforts associated with this clinical trial.  Should you have any questions regarding this post, please feel free to contact the NNPDF Central Offices at:  nnpdf@nnpdf.org or the foundation web site at:  www.nnpdf.org .  In addition, for a complete historical timeline on the Cyclodextrin Clinical trial, please refer to the NNPDF’s Cyclodextrin web page for more details.

[01/21/2014 ~ blg]

Div

Nov

Dear NNPDF Families and Friends,

The NNPDF January 2015 e-Newsletter is available! Highlights include:

  • Clinical Trial News and Updates
  • Infiniti Coaches Charity Challenge
  • 2015 NNPDF Board Meeting

Click here to view the NNPDF January 2015 e-Newsletter!

[Jan 20th, 2015 ~ blg]


Banner

Dear NNPDF Families and Friends,

RE:  Orphazyme ~ "Orphazyme ApS (www.orphazyme.com) was founded in June 2009 with the aim of developing heat shock protein based therapy for the treatment of diseases caused by defects in the function and/or metabolism of proteins."

The NNPDF is making press release announcements from Orphazyme, pertaining to their advances towards a clinical trial for Niemann-Pick Disease type C, available to the wider NPC Community.

Listed below are the links back to the Orphazyme APS (LLC) website to read the press releases.

Orphazyme Raised 20 Million Euros in a Series B Financing Round
01/09/2015
COMP Issues Prositive Opinion on Orphan Drug Application for Arimoclomol
10/20/2014
Orphazyme in Final Preparation Stages for a Phase II/III Clinical Trial  
10/06/2014

For a full archive of Orphazyme updates, visit the NNPDF Orphazyme web page

“The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet."

[Jan 15th, 2015 ~ blg]


Vtesse

~Cyclodextrin Update~ 01/07/2015
Update on NPC Cyclodextrin Trial

Leading Life Science Syndicate Commits $25 Million to Series A Funding to Launch Vtesse, Inc., the First Rare Disease Company Spun Out of Cydan Development, Inc.

Additional Rersources:

WSJ
Wall Street Journal (WSJ) Article

Small Biotech Gets Rights to Rare Disease Drug
(paid subscription required to read)

 

Dear NNPDF Families and Friends,

We are pleased to share with the foundations Niemann-Pick Disease type C (NPC) families and community a recent development pertaining to the Cyclodextrin clinical trial. The recently incorporated, Vtesse, a rare disease company spun-off from Cydan Development, Inc., which is focused on developing drugs for Niemann-Pick Disease Type C (NPC) and other severe diseases with great unmet need, will begin collaborating with the National Institutes of Health on furthering development of Cyclodextrin (VTS-0270) for Niemann-Pick type C.

Vtesse also announced that it has established a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and the National Center for Advancing Translational Sciences (NCATS), each a component of the National Institutes of Health (NIH). Vtesse and NCATS have also entered into a licensing agreement for the current rights held by NIH for the worldwide use of cyclodextrin, delta-tocopherol, and derivatives of tocopherol, alone or in combination, for the treatment of lysosomal storage diseases (LSDs), including NPC. Regulatory orphan designations for the U.S. and EU will be also be transferred to Vtesse.

Vtesse will use the proceeds from the $25 Million raised through its Series A financing to conduct a clinical program for VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for NPC, and to discover and pre-clinically evaluate additional novel drugs for NPC and other lysosomal storage diseases.

For more information on phase 2 & 3 of the Cyclodextrin clinical trials & the transition from NIH to Vtesse, Inc, please review the full press release here: Click Here

The NNPDF Central Offices will continue to assist with the dissemination of information pertaining to the news and updates relating to the ongoing efforts associated with this clinical trial.  Should you have any questions regarding this post, please feel free to contact the NNPDF Central Offices at:  nnpdf@nnpdf.org or the foundation web site at:  www.nnpdf.org .  In addition, for a complete historical timeline on the Cyclodextrin Clinical trial, please refer to the NNPDF’s Cyclodextrin web page for more details.

[Jan 7th, 2015 ~ blg]

 


 

Elisha Elaine Kudrna
04/19/1994 ~ 11/12/2014

 

Dear NNPDF Families and Friends,

It is with heavy hearts that the NNPDF central office notify the foundation membership of Elisha Elaine Kudrna's passing. Daughter of Jeff & Monica Kudrna & beloved sister to brother Daniel, Elisha lost her battle with Niemann-Pick type A/B on November 12th, 2014.  She passed away at home, surrounded by her family at the age of 20.

Elisha's mother, Monica, wrote to us in memory of her beautiful daughter:

"She was a bright star in our family, and now she shines eternally in heaven.

She endured so many obstacles in her life, but none of them did she not conquer without a smile. With our faith in God, we know that Elisha had a full purpose in this life, and she is now in heaven with a healed body, dancing and singing loudly.  As we miss her with aching hearts every day, we are reassured that we will see her again."

Our loving support and hearts go out to the Kudrna family as they move ahead. Please know that your NPD family and community grieves with you ~ and ~ will be keeping all who loved Elisha and held her dear in our thoughts and hearts in the days, weeks and months ahead.

 

[Jan 6th, 2015 ~ blg]

 



Dear NNPDF Families and Friends,

Today, as we all take time to reflect on the past year, the NNPDF community has quite a number of uplifting and positive milestones upon which to reflect  for 2014.  Unfortunately, we again have had heartbreaking losses of loved ones to Niemann-Pick Disease whom we all hold so close to our hearts.  Despite these soul crushing losses, the NPD community has made considerable progress in research and advancement towards therapies with the start of patient clinical trials for both Niemann-Pick type C and ASMD (NPD Type B).  There have also been many personal triumphs shared within the community and the foundation continually to strives towards offering and building upon our stellar family support programs and continued financial commitments towards research.

 

We have been fortunate to be a part of your lives throughout the year and are looking forward to the HOPE which 2015 will bring.

Have a warm and love filled New Years and we'll see you in 2015!

[Jan 1st, 2015 ~ blg]



Newsline postings from 2014 & 2013

NewsLine postings from 2012, 2011, 2010 and 2009              NewsLine postings from 2008 and prior

"The Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) does not engage in the practice of medicine..  It is not a medical authority nor does it claim to have medical knowledge.  This site is an educational service of the Canadian Chapter of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice.  Information contained or suggested on this Web site does not constitute medical advice.  For all information related to care, medication or treatment, the CCNNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet.”