Families of Spinal Muscular Atrophy Awards 7 New Basic Research Grants

Families of SMA Awards 7 New Basic Research Grants for $745,000 to Develop New Approaches for SMA Therapies. 

October 18, 2011.

In the Fall 2011 issue of “Compass” Families of Spinal Muscular Atrophy announces seven new basic research grant awards. These new awards encompass $745,000 for the basic research portion of the $3 Million in new research funding that FSMA announced earlier this year.

Click the image below to download a copy and read a description of our newly funded projects:

What is Our Research Goal at FSMA?
Our main goal is to accelerate the discovery of an effective therapy and cure for SMA by funding and advancing a comprehensive research program, using a three-pronged approach:
1) Basic Research to reveal the best ways of making SMA drugs,
2) Drug Discovery to make new drugs, and
3) Developing Clinical Trial infrastructure to help test new drugs.

Why is Basic Research Important to Our Overall Strategy?
Basic research is a critical component in finding a treatment for SMA. It provides fundamental information about what is going wrong in SMA by telling us when and where SMN protein is needed, and what SMN protein does in different cell types. This knowledge provides seed ideas for new ways of making drugs.

The FSMA basic research program is governed by our Scientific Advisory Board (SAB). SAB bios can be found by clicking here. The SAB carefully reviews all grant applications to ensure that we fund only the best quality research relevant to our mission.

What’s Exciting About Our New Research Grant Awards?
Three of the seven new grants for 2011 will help us to understand why motor neurons are selectively vulnerable to lowered SMN levels compared to other tissue types. These grants include projects led by Dr. Henderson at Columbia University, Dr. Rossoll at Emory University, and Dr. Kothary at the University of Ottawa.

The grant to Dr. Ko at the University of Southern California will help us understand exactly where defects in SMA occur and the reasons for observed selectivity at different muscles. The grant to Dr. Monani at Columbia University will help identify new genes that are protective against lowered SMN levels and will lead to new drug targets. Finally the funded projects led by Dr. Kolb at The Ohio State University and Dr. Simard at the University of Manitoba will validate molecular biomarkers that could make future clinical trials more efficient.

Since 2004, the FSMA Scientific Advisory Board has awarded 66 basic research grants for a total of $8,195,096 in funding.  This is in addition to our drug discovery and clinical research funding.  The grants have been awarded to 47 different principal investigators at 33 different institutions. 

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