Welcome to the Canadian Chapter of the National Niemann-Pick Disease Foundation Website

The Canadian Chapter of 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.

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

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


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

~ 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

The Canadian Chapter of the National Niemann-Pick Disease Foundation was established in April 2005.

 The primary goals of the foundation are:

Our Web site can help you learn more about Niemann-Pick Disease. The NewsLine, Research, and Media pages are resources for the latest in NPD news. Meet our board of directors in the About Us section. Click here to read an introductory letter from Tammy Vaughan, Chair of the Canadian Chapter of the National Niemann- Pick Disease Foundation. Feel free to send us an email as we enjoy getting feedback from our visitors. In the Events section you'll find write-ups and photos from our fundraising events. For donation information, please visit the Donate Now page.

Thank you for visiting the CCNNPDF Web site, and please let us know if we can be of any assistance!

Foundation News:


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International Niemann-Pick Disease Organizations

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Research News & Announcements:

Dr Marc Patterson
Mayo Clinic Podcast
featuring Dr Marc Patterson
speaking about
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

National Niemann-Pick Disease Foundation (NNPDF)(U.S.)

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.  For more information visit

Mia's NPA Timeline
Mia Walts, NPA
5/31/09 - 10/29/11

"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.”

This site does not host or receive funding from advertising or from the display of commercial content.

The Canadian Chapter of the National Niemann-Pick Disease Foundation is a registered charitable organization.  Your donations are fully tax-deductible.

Web Site updated 08/14/2014 ~ blg

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