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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 Vision...is 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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NNPDF Clinical Trials: News & Updates

NIH Cyclodextrin Trial
For Niemann-Pick Disease Type C ~ Pediatrics
~ UPDATE ~
Dateline: 6/ 27/2014

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

 

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ALERT: NOW RECRUITING

NIH  Clinical Trial Updates for NPC Adults
HDAC Inhibitor ~ vorinostat
Dateline: 09/12/2014

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For an update from Dr. Forbes D. Porter to share with the community in regards to recent developments in both the Cyclodextrin and new Histone Deacetylase Inhibitors (HDACi) ~ vorinostat clinical trials.

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Niemann-Pick Disease Type B Adults
Genzyme ERT Clinical Trial Update
Dateline: 7/13/2014

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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

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For the Updates

Orphazyme Clinical Trial Update for Niemann-Pick C
Dateline: 09/08/2014

The National Niemann-Pick Disease Foundation is pleased to share an announcement received from Orphazyme ApS of an upcoming clinical trial for Niemann-Pick Disease Type C.  Orphazyme ApS (Copenhagen, Denmark) develops new therapies for the treatment of rare and genetic diseases. 

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Visit the Orphazyme Clinical Trial Page!


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National Niemann-Pick Disease Foundation

~ Research Strategy ~

In 2014, the National Niemann-Pick Disease Foundation Board reviewed its research funding strategy and has produced a new strategic plan for research.

Account was taken of the needs of our Membership; current knowledge in the field of Niemann-Pick Disease; the funding strategies of other NPD organizations; and the advice and expertise of the members of the NNPDF's Scientific Advisory Board.

As a result we are pleased to announce that the NNPDF will fund Pre-Doctoral, Post-Doctoral and Early career investigator fellowships in all areas of promise - basic, translational and clinical research- with regards to Niemann Pick Disease.

The NNPDF strives to enable promising new researchers to make a career in the field of Niemann-Pick Disease, thus providing for a new generation of scientists to accelerate the pace of NPD research.

"The NPD community is one of the most collaborative there is. The level of idea/data sharing among scientists is remarkable. We are also a model for other rare diseases with respect to our ability to accelerate work at the bench to therapeutics. And much of this has been made possible by the family-researcher partnerships that have sustained this work over the past 20 years." - Dr. Dan Ory, Current NNPDF Scientific Advisory Board member & past SAB Chair (Sept. 2007 ~ Jan. 2014)

A key element to the foundation’s research strategy is the ability to track funds raised within the communities of our family membership.  Great care is given to identify monies received at the NNPDF Central Offices through family fundraising efforts so that a specific designation by disease type and a restriction towards research can be made.  The NNPDF is proud to ensure our family membership that 100% of the funds raised through their community events are directed towards NPD research

Current NNPDF Funded Grants & Fellowships

WSJ

A Wall Street Journal Exclusive!


Trials: A Desperate Fight to Save Kids & Change Science
by Amy Dockser Marcus
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Watch the Memorial Video presented at the NNPDF Family Conference in San Francisco, California.

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Foundation Newsline
Quick Links

 

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Here for you Always
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Song written for the NNPDF by Rachel Taylor, cousin of Lorna Tyrell (mom of Naomi, NPC)

Coming Soon...
Think Again. Think NPC. Campaign in the United States

NNPDF is working colaboratively with the International NIemann-Pick Disease Alliance (INPDA) to raise awareness within the healthcare professional community. A major hurdle that we learn about time and again from our NNPDF membership is the challenge to a obtain a correct diagnosis of Niemann-Pick type C Disease (NP-C).  Due to this, NP-C takes, on average, five years to diagnose.  Because Niemann-Pick type C is treatable this means that patients live for five years without treatment or access to support.

Follow these links as the NNPDF will be developing more information in the months to come.

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Click here to view a video explaining the program


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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 www.nnpdf.ca

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  10/22/2014 ~ blg

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