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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Lysosomal Storage Disease Research Consortium

In 2005-2006, NTSAD focused its research funding on the Lysosomal Storage Disease Research Consortium (LSDRC) grants initiative. Co-founded by NTSAD and the National MPS Society in 2005, the LSDRC was a collaborative research-funding group made up of patient advocacy groups representing lysosomal storage diseases (LSDs). The LSDRC entered into an agreement with the National Institute of Neurological Disorders and Stroke (NINDS) to establish a three-year, jointly sponsored program to provide financial, scientific and administrative support towards preclinical or translational research specifically addressing the neurological aspects of lysosomal storage disorders.

The goal of this program was to stimulate interest in and to solicit applications for lysosomal storage disorder research focused on improving central nervous system (CNS) treatment outcomes, enhancing the effectiveness of delivery and targeting of cells, enzymes, drugs and genes into the brain. This funding opportunity specifically encourages the transition from basic studies in LSDs to translational research for improved delivery of therapeutic cells, proteins, genes and small molecules across the blood-brain barrier. 

The Review Committee prioritized applications needing additional targeted research that would improve the principal investigator's opportunity of receiving NIH funding. Thirteen investigators received a total of $360K in LSDRC grants over the period 2006-2008. Of these investigators, six of them received NIH follow-on grants which is a higher than average success rate.

The LSDRC patient advocacy group partners were as follows:

  pdf LSDRC Grants Funded 2006 - 2008 (69 KB)  (.pdf)