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January 2017 Newsletter



Rare Disease Day 2017

Research brings hope to people living with rare diseases.

People with a rare disease such as Niemann-Pick Disease have tremendous challenges which can include a prolonged time in receiving a correct diagnosis, finding support resources, finding appropriate quality health care, and difficulties in accessing available treatment. This often results in heavy social and financial burdens on patients.

Rare Disease Day® is an annual event taking place on the last day of February each year. The main objective is to raise awareness about rare diseases and their impact on patients’ lives. Many events are hosted to raise awareness for Rare Disease Day throughout the month of February and on Rare Disease Day. Please visit to learn what’s happening around the world. The National Organization for Rare Diseases (NORD) is the official U.S. Sponsor of Rare Disease Day. The global event was founded by EURORDIS, a partner of NORD.

Anyone can get involved in Rare Disease Day awareness.  U.S. participants can find helpful resources and activities to get involved and events taking place near them at

NNPDF Rare Disease Day Participation

To raise awareness for Niemann-Pick Disease and Rare Disease Day on February 28, 2017, the NNPDF central office staff will be offering customized Facebook profile pictures. Email your Facebook profile picture request to Jill at

In your request please include;

  1.  A photo of your NPD loved one
  2.  In Honor of or In Memory of
  3.  Your loved ones name as you would like it to appear
  4.  Niemann-Pick Disease type

Along with your custom Facebook profile picture we will include our Facebook banner for your use.




For the most current NPD developments, subscribe to our NNPDF Blog!

All updates, press releases, and announcements
are PRIMARILY posted on our blog!


DON’T MISS OUT ON IMPORTANT INFORMATION! Sign up now and receive an email notice as soon as an update is posted on our NNPDF Blog. This is a free and convenient service provided to our NPD community and subscribing is easy!

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Upcoming Fundraisers


Yankee Candle Online Fundraiser
In Memory of Bryanna Desouza and
In Honor of Niemann-Pick Disease Families

February 13, 2017 – June 30, 2017

Bryanna Desouza (NPC)  |  Jan 20, 1992 ~ Dec 5, 2006

Judy Desouza will be hosting a Yankee Candle fundraiser once again, in memory of her daughter, Bryanna (NPC) and in honor of all Niemann-Pick Disease families. Judy, in working with Yankee Candles, will dedicate 40% of profits from both local and online orders to the NNPDF.



Miracle for Milana Benefit

Friday, January 20, 2017
Auletto Caterers  |  1849 Cooper St, Almonesson  |  New Jersey

Join us for an evening of food, drinks, entertainment, Chinese auction, and much more. Please join us and together we can give Milana the best life possible and fight together to cure this horrific disease for her and for all other NPC families.



Infiniti Coaches’ Charity Challenge

A vote for coach Painter is a vote to Help Fight NPC!


Infiniti and the National Association of Basketball Coaches is once again sponsoring the Infiniti Coaches’ Charity Challenge. In its seventh year, the Infiniti Coaches’ Charity Challenge rivals 48 NCAA Division 1 basketball coaches to compete by online voting to win $100,000 for the charity of their choice.

Purdue University Coach Matt Painter has once again named the BReaK Thru Fund for NPC Research as his charity! The BReaK Thru Fund for NPC Research was started by the Smith family who lost 3 children to NPC.

The Infiniti Coaches’ Charity Challenge program consists of a partnership between Infiniti, the National Association of Basketball Coaches (NABC), the NCAA, and ESPN. It has grown to include 48 NCAA Division I men’s basketball coaches.

VOTE COACH PAINTER daily until March 11, 2017 and help raise $100,000 for NPC Research through the Smith Family BReaK Thru Fund.



From the Wylder Nation Foundation


Exciting news for Niemann-Pick Type A patients shared from the Wylder Nation Foundation. Perlara PBC and Biovista are both making progress in discovering treatment options for Niemann-Pick Type A.

Follow the links below for the latest announcements.



Dr. Andrew Munkasci
Published Paper on Vorinostat

The NNPDF Central Office has received a copy of a recent publication by  Dr. Andrew Munkacsi, an NNPDF/CCNNPDF funded fellow, that provides insight into Vorinostat as a candidate therapy to treat NPC Disease via the first animal study in a model of NPC disease. 

To view the publication abstract and complete publication
follow the links below.



Research Updates

Following are the latest research updates received in the NNPDF Central Office.

. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 

Sanofi Genzyme UCSF Clinical Trial Site Now Open

. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 

Vtesse Announces Dosing of First Patient in Australia in
Phase 2b/3 Clinical Trial of VTS-270 in Niemann-Pick Type C1 Disease
Vtesse December Newsletters in Several Languages
Vtesse Announces Appointment of
Jason Meyenburg as Chief Commercial Officer

. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 

AIDNPC Clinical Programme Update #12
in Several Languages

. . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 

FDA Grants Fast Track Designation for
Trappsol (R) Cyclo(TM) in
Drug Development Program for NPC

CTD Holdings Files Clinical Trials Application
to Continue Advancing Trappsol® Cyco™
Drug Development Program to Treat NPC


9 8.16

PO Box 49, Fort Atkinson, WI 53538-0049
Phone: 920-563-0930  |  Toll Free: 877-287-3672 |



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