Welcome to the National Niemann-Pick Disease Foundation Website

The National Niemann-Pick Disease Foundation supports and promotes research to find treatments and a cure for all types of Niemann-Pick Disease, and we provide support services for individuals and families affected by NPD. Our a world where Niemann-Pick Disease is no longer a threat to a full and productive life for patients and their families.

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22nd Annual NNPDF Family Support and Medical Conference  

Thursday, July 31st ~ Sunday, August 3rd, 2014


Hotel, Family Conference Registration and Helping Hand Stipend now available online!

Genzyme ERT Clinical Trial Update
JUNE 27th, 2014


Left to right, Alan Gilstrap, Director, Advocacy Development - Rare Disease; Genzyme Corporation  ; Sandra Cowie, NNPDF Research Committee Chairperson ; Dr. Simon Jones, Manchester Centre for Genomic Medicine St. Mary's Hospital, University of Manchester, England; Nadine Hill, NNPDF Executive Director

For the Updates

NIH Cyclodextrin Trial
JUNE 27th, 2014

We received the following update from Dr. Denny Porter with reference to the NIH/TRND Cyclodextrin Clinical





A Wall Street Journal Exclusive!

Trials: A Desperate Fight to Save Kids & Change Science
by Amy Dockser Marcus

Click HERE

Rare Diseases Research:
Clinical Trial for Niemann-Pick Type C

Click to watch the video

Determined to find a treatment for children with the degenerative brain disease Niemann-Pick Type C, Steven Walkley, D.V.M., Ph.D., turned a serendipitous laboratory discovery into a successful national research collaboration with other academic institutions and the National Center for Advancing Translational Sciences' program for rare diseases (Therapeutics for Rare and Neglected Diseases). 

These efforts led to an NIH Phase 1 clinical trial testing cyclodextrin 
as a therapy for children with this disease.

scientist in lab

Niemann-Pick Disease Research

~ JUNE 2014 ~
Denny Porter

Dr. Forbes "Denny" Porter
National Institutes of Health NPC Clinical Coordinator

Update on NPC Cyclodextrin
Clinical Trial

(NEW ~ 06/27/14)

NIH Update from HDACi
Clinical Trial

(NEW ~ 06/27/14)
NIH Update on NPC Cyclodextrin Clinical Trial

Update from Genzyme on ASMD Development Efforts


Foundation Newsline
Quick Links


The 2014 NNPDF Board of Director's  will be meeting in San Francisco, CA on Thursday, July 31st

at 9:00 AM.  The NNPDF board meetings are open to all members of the NNPDF. Please refer to

the NNPDF Family Conference to be held in San Francisco, CA for more inforamtion ~ or ~ contact

the NNPDF Central Offices at: if you are interested in attending.  You may learn more about the

current members of the Board of Director's at: Meet the NNPDF BoD's

Foundation News:

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earth graphic
International Niemann-Pick Disease Organizations

Research News & Announcements:

Dr Marc Patterson
Mayo Clinic Podcast
featuring Dr Marc Patterson
on Niemann-Pick Type C.

Family Resources & Support:

The Progression of NPC in Two Children's Lives

Adam three years old
Adam Ward, NPC
1/13/90 - 6/4/00
Stacey Vorpahl
Stacey Vorpahl, NPC
1/3/85 - 10/9/04
Mia's NPA Timeline
Mia Walts, NPA
5/31/09 - 10/29/11
Mia Walts

“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.?

This Web site is a service of the National Niemann-Pick Disease Foundation's Family Services Program, made possible by the financial support of our member families and other generous donors and grantors. This site receives no government funding, nor does it host or receive funding from advertising or from the display of commercial content.

This site complies to the HONcode standard for trustworthy health information: verify here.

The National Niemann-Pick Disease Foundation is a 501(c)(3) organization.  Your donations are fully tax-deductible.

Web Site updated  07/24/2014 ~ blg

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