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

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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. The FSMA basic research program is governed by the Scientific Advisory Board (SAB). The SAB carefully reviews all grant applications to ensure funding of only the best quality research relevant to the mission of Families of SMA.

Three of the seven new grants for 2011 will help the understanding of 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 the understanding of 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.

Detailed descriptions of each project can be found at http://www.curesma.org

The main goal for Families of SMA research programs 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.

Basic research is a critical component in finding a treatment for SMA. It provides fundamental information about what is going wrong in SMA by indicating 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 the Scientific Advisory Board (SAB). The SAB carefully reviews all grant applications to ensure funding of only the best quality research relevant to the mission of Families of SMA.

The newly funded projects include:

Stem Cell Models of SMA: Molecular and Cellular Mechanisms
Christopher Henderson, Ph.D., Columbia University, $160,000 for 2 years.

New Neuromuscular Preparations for In Vivo Evaluations of Drug Efficacy in SMA
Chien-Ping Ko, Ph.D., University of Southern California, $70,000 for 1 year.

Validation of Spinal Muscular Atrophy Biomarkers in VALIANT Subjects
Stephen Kolb, M.D., Ph.D., The Ohio State University, $70,000 for 1 year.
Louise Simard, Ph.D., University of Manitoba, $25,000 for 1 year.

Identification and Characterization of Factors Critical in Regulating the Selective Vulnerability of Distinct Motor Neuron Pools in SMA Model Mice
Rashmi Kothary, Ph.D., Lindsay Murray, Ph.D., University of Ottawa, $120,000 for 2 years.

Exploring novel genetic determinants of disease severity in spinal muscular atrophy model mice.
Umrao Monani, Ph.D., Columbia University, $160,000 for 2 years.

Effects of SMN on mRNA transport and local protein synthesis in motor axons. Wilfried Rossoll, Ph.D., Claudia Fallini, Ph.D., Emory University, $140,000 for 2 years.

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 funding for drug discovery and clinical research funding. The grants have been awarded to 47 different principal investigators at 33 different institutions.

About Families of Spinal Muscular Atrophy:
FSMA is dedicated to creating a treatment and cure by: Funding and advancing a comprehensive research program; Supporting SMA families through networking, information and services; Improving care for all SMA patients; Educating health professionals and the public about SMA; Enlisting government support, and; Embracing all touched by SMA in a caring community. http://www.curesma.org

Families of SMA funds and directs the leading SMA research programs. The successful results and progress from basic research to drug discovery programs to clinical trials provides real hope for families and patients. Families of SMA has funded 5 leading new drug development programs for therapies specially designed to treat SMA.

FSMA is a non-profit, 501(c)3 tax exempt organization with 30 Chapters throughout the United States and over 70,000 members and supporters.

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