SMA Foundation Funds Over $15 Million to Develop Treatments for SMA

New York, NY – November 29, 2004 – 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 MIT / The Whitehead Institute
Albert Einstein College of Medicine Northwestern University
American Academy of Neurology The Ohio State University
Children’s Hospital Boston PsychoGenics, Inc.
The Children’s Hospital of Philadelphia Stanford University
Cold Spring Harbor Laboratory Texas Scottish Rite Hospital for Children
Columbia University Medical Center University of Rochester
CombinatoRx University of Utah
Curis, Inc. University of Würzberg – Germany
The Jackson Laboratory 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 or call (646) 253-7100.

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Media Contact:
Bryan deCastro
(631) 495-9177
[email protected]

Cynthia Joyce
(646) 253-7100
[email protected]