Tuesday, November 27, 2012

Fellowship Announcement

National Niemann-Pick Disease Foundation Research Post-Doctoral Fellowship Funding Announcement 

~Oct/Nov 2012~ 

The National Niemann Pick Disease Foundation (NNPDF) has chosen to fund post doctoral fellowships for the past several years. These fellowships support a post doctoral fellow to work on a research project in the field of Niemann-Pick Disease for a period of two years. The NNPDF has utilized strategies to allocate funds raised by NPD families by disease type to support these specific NPD disease focused fellowships.

In the past several years, NNPDF has been able to fund a number of fellowships for Niemann-Pick Disease Type C. These are called the Peter G. Pentchev Research Fellowships and the NNPDF in conjunction with the Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) are currently funding the work of five (5) Peter Pentchev fellowships.

This year we were able to establish a fellowship for Acid Sphingomyelinase Deficiency (ASMD; i.e., Types A and B Niemann-Pick Disease) research entitled the Edward H. Schuchman Research Fellowship for ASMD Niemann Pick Disease. We received 5 strong applications for the fellowship and I am pleased to announce that the first Edward H. Schuchman Fellowship has been awarded to Dr. Lluis Samaranch Gusi (sponsor Dr. Krystof S. Bankiewicz, MD, Ph.D.) at the University of California San Francisco.

This fellowship research project is entitled: “AAV9 mediated human acid sphingomyelinase expression in the non-human primate brain; Preclinical development of gene therapy for Niemann-Pick Disease Type A”. The goal of this project is to develop a treatment for the neurological consequences of Niemann-Pick Type A Disease. This project will evaluate the feasibility of delivering an adeno-associated virus encoding human acid sphingomyelinase globally into the brain by injection of a vector into the brain and/or the spinal canal.

Respectfully submitted by:
Sandra Cowie
NNPDF Research Committee Co-chair

For more detailed information please Click here.

For information on the history of Research funding you can visit the following pages:

[Nov. 27, 2012 blg]

Thursday, November 15, 2012

NIH Cyclodextrin Trial Updates

Hello NNPDF Families and Friends, 

We have received an additional update from the Therapeutics for Rare and Neglected Diseases ~ Niemann-Pick Disease Type C Team (TRND NPC Team) at the National Institutes of Health (NIH) with new information on the Phase I Study of the Cyclodextrin (HP-b-CD) for NPC1 Disease. 

The application for an Investigational New Drug (IND) was filed with the United States Food and Drug Administration (FDA) on Wednesday, November 14th, 2012! Please follow the link below to the NNPDF "Cyclodextrin" web page for more details. 

Sincere and heartfelt THANKS to ALL those who have worked to diligently to see this program move forward. We WILL Persevere in our Quest for a Cure!