Thursday, December 31, 2015

We Will Persevere!


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

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!

Wednesday, December 30, 2015

Giving the Gift of Hope!

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!
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 find out how you can help give the Gift of Hope, click here for the e-Blast:

Tuesday, December 22, 2015

Season's Greetings from the NNPDF!


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

Monday, December 21, 2015

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

Friday, December 18, 2015

Dec 18th, 2015 ~ 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: 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

Wednesday, December 16, 2015

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


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.

Tuesday, December 15, 2015

Year-end Shopping for the Gift of Hope!


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:

Wednesday, December 9, 2015

ALERT: New Recruiting Sites for VTS-270 (2-hydroxypropyl-β-cyclodextrin) for NPC

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 page. To view these criteria, location details and for additional contact information please visit 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:
NIH Phase 1 Clinical Trial Historical Timeline can be viewed:

Tuesday, December 8, 2015

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


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.

A link to Kaitlyn’s obituary will be posted as soon as it is available on the NNPDF website:

Monday, December 7, 2015

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


Register: US and Canadian Audience, 12:30 PM EST*, December 14, 2015

*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:


Nadine M. Hill

Executive Director
National Niemann-Pick Disease Foundation

Vtesse Phase 2b/3 Clinical Trial Historical Timeline can be viewed:
NIH Phase 1 Clinical Trial Historical Timeline can be viewed:

Friday, December 4, 2015

Special Update from the NNPDF Board of Directors ~ December 4th, 2015


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 for the most recent update to the NNPDF membership; dated: Dec 4th, 2015 and or follow this direct link:

Tuesday, December 1, 2015

Today is the Day! ~ #GivingHopeTuesday

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!
1. Simply make a #GivingHOPETuesday financial gift online today ~ via the Donate NOW link.
2. Consider making your year-end tax-deductible donation to the NNPDF today, 12/1/15 ~ your dollars will earn matching funds!!
3. DOUBLE DONATION DAY at Bravelets! 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!
4. Check out our #GivingTuesday e-News blast for more ways to give back this holiday shopping season!
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!