The Spinal Muscular Atrophy Foundation, a nonprofit organization dedicated to finding a treatment or cure for spinal muscular atrophy (SMA) – the leading genetic killer of infants and toddlers – announced that is has funded more than $15 million in research in the last 18 months. Projects were selected from a large pool of industry and academic investigators on the basis of scientific promise and potential to advance therapeutics development. The Foundation continues to seek out innovative and high quality research opportunities particularly those in the neuroscience biotechnology sector.

Spinal Muscular Atrophy is a genetic motor neuron disease caused by the progressive degeneration of nerve cells in the spinal cord and brainstem, leading to muscle weakness, respiratory complications and premature death.

Having been identified by researchers as the neurological disease closest to a treatment, SMA has been selected by the National Institute of Neurological Disorders and Stroke (NINDS) to serve as the prototype for a translational research project that is expected to yield drug candidates for Investigational New Drug Application (IND) filings within three to five years. The SMA Foundation estimates that there are currently 55,000 people suffering from SMA in the United States, Europe and Japan and that a conservative annual market potential for an SMA treatment could exceed $500 million.

The SMA Foundation has been a major catalyst in the fight against this disease and has demonstrated their commitment to finding a treatment or cure by providing the necessary funding to advance cutting edge research. Funded projects span the spectrum of basic, translational clinical research.

In 2004, the SMA Foundation forged a number of biotech partnerships – two of the larger transactions with Curis, Inc. and CombinatoRx were each valued in the single-digit, million dollar range. For 2005, the Foundation will place an increasing emphasis on drug development efforts with the biotechnology industry. The Foundation continues to fund strong academic research endeavors that have a high use value to translational research.

The following organizations have received funding:

Academia Sinica – Taiwan
Albert Einstein College of Medicine
American Academy of Neurology
Children’s Hospital Boston
The Children’s Hospital of Philadelphia
Cold Spring Harbor Laboratory
Columbia University Medical Center
CombinatoRx
Curis, Inc.
The Jackson Laboratory
MIT / The Whitehead Institute
Northwestern University
The Ohio State University
PsychoGenics, Inc.
Stanford University
Texas Scottish Rite Hospital for Children
University of Rochester
University of Utah
University of Würzberg – Germany
Washington University

About SMA Foundation
The SMA Foundation is a nonprofit organization founded in 2003 dedicated to finding a treatment and potential cure for Spinal Muscular Atrophy (SMA). The Foundation provides funding for the full range of research from basic to clinical work conducted in academic laboratories as well as corporate therapeutics development. In addition, the Foundation is committed to raising awareness, education and increased federal funding and support. For more information on the Spinal Muscular Atrophy Foundation, visit www.smafoundation.org or call (646) 253-7100.

Media Contact:
Bryan deCastro, (631) 495-9177
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Cynthia Joyce, (646) 253-7100
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