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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The Families and Friends of the NNPDF took time during the Family Conference in San Francisco, CA to remember those loved ones who passed away from Niemann-Pick Disease in the last year.


Click here to watch the Memorial Presentation Video presented at the
NNPDF Family Conference in San Francisco, CA.

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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

Dr. Edward Schuchman Recognized for  Work on NPD (Type A & B ~ ASMD)


Sandra Cowie - NNPDF Research Committee Chair & Dr. Edward H. Schuchman - NNPDF Scientific Advisory Board Member

Visit the Newsline to read the article.


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

Foundation News:

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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:

CCNNPDF logo Canadian Chapter National Niemann-Pick Disease Foundation
The CCNNPDF works with the NNPDF to promote research, provide education, information and referrals, and to support families who have received a diagnosis of Niemann-Pick Disease.  Visit

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  08/29/2014 ~ blg

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