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Current and Recent Research Grants

The research funded by the NNPDF is made possible in great part due to the efforts of our member families and their extended
support networks via local community-sponsored events, and to the support of the Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF). The NNPDF is truly grateful for this support!

Research Grants jointly sponsored by the NNPDF and CCNNPDF
Fellowships jointly sponsored by the NNPDF and CCNNPDF
Clinical Research and Services funded by the NNPDF


Postdoctoral Research Fellowships


The NNPDF and the CCNNPDF are pleased to be able to jointly sponsor the Peter G. Pentchev Research Fellowships. 
Visit the Funded Fellowships page for links to Project Summaries and updates about these research projects.

Dr. Andrew Munkacsi
Defining the molecular basis of HDAC inhibitors to treat murine, feline, and human models of NPC
Sponsored by Stephen L. Sturley, Ph.D., Columbia University Medical Center; New York, New York; USA
Dr. Munkacsi's fellowship began September 2011.

Dr. Nicholas Cianciola
Activation of an alternative cholesterol homeostatic mechanism in NPC
Sponsored by Cathleen Carlin, Ph.D., School of Medicine, Case Western Reserve University; Cleveland, Ohio; USA
Two years September 2010 - September 2012  $100,000 (funding extended a third year to September 2013)

Dr. Dorothea Maetzel
In Vitro modelling of Niemann-Pick type C Disease Using Patient-Specific Induced Pluripotent Stem Cells
Sponsored by Rudolf Jaenisch, M.D., Whitehead Institute for Biomedical Research; Cambridge, Massachusetts; USA
Two years September 2010 - September 2012   $100,000 (funding extended a third year to September 2013)

Dr. Fabrizio Vacca
Analysis of cholesterol export from purified endosomes in NPC cellular models
Sponsored by Professor Jean Gruenberg, University of Geneva, Switzerland.
Two years April 2010 – April 2012   $100,000

Dr. Ian Martin Williams
Neurobiology of Purkinje Cells in NPC1
Sponsored by Professor Fran Platt, University of Oxford, UK
Two years September 2009 – September 2011   $100,000

[Nov 2, 2010 mem; Nov 28, 2011 mem; Mar 13, 2012 mem]


Mouse Model Grant to Jackson Laboratory

The NNPDF is pleased be able to provide $2500 to help defray the costs of processing the mouse model related to Niemann-Pick Disease.  The mouse model was created by Dr. Richard Pagano of the Mayo Clinic College of Medicine and is being processed by the Jackson Laboratory Repository.  

The nonprofit Jackson Laboratory Repository in Bar Harbor, Maine, serves as a centralized NIH-funded facility for mouse models used by the international biomedical research community, in both academic and nonprofit institutions. 

[Jan 20, 2010 mem]


Edward H. Schuchman, Ph.D. (Mount Sinai School of Medicine). Construction & Characterization of New Mouse Models for Types A & B Niemann-Pick.   Click here to read a September 2008 progress summary.    Click here to read the May 2009 progress summary.

Frances M. Platt, Ph.D. (Dept. of Pharmacology, University of Oxford).  Targeting sphingosine storage as a new therapeutic approach for Niemann-Pick Type C.  Click here to read the October 2008 progress summary.   Click here to read the March 2009 progress summary.      Click here to read the October 2009 progress summary.

Seymour Packman, MD, Director, UCSF-Stanford Lysosomal Disease Center
Underwritten by a grant from Genzyme Corporation

(To see a listing of Fellowships funded by the NNPDF, click here.)

(To see a listing of Research Grants funded by the NNPDF, click here.)

(To see a listing of Clinical Research and Services funded by the NNPDF, click here.)

[June 22, 2009 mem]

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