Statistics
- SMA is the #1 genetic killer of young children;
- SMA is estimated to occur in nearly 1 out of every 6,000 births;
- 1 in every 35 people, or nearly 10 million Americans, unknowingly carries the gene deletion responsible for SMA; few have any known family history;
- SMA is a pan-ethnic disease and does not discriminate based on race, ethnicity, or gender;
- there is currently no treatment and no cure, but the National Institutes of Health (NIH) selected SMA as the disease closest to treatment of more than 600 neurological disorders;
- researchers estimate that a viable treatment and/or cure is attainable in as little as 5 years – if provided adequate resources;
- the American College of Medical Genetics recommends that SMA carrier testing be made available to all couples planning a family, regardless of ethnicity or family history.

This site is aimed at everyone involved in the fight against spinal muscular atrophy, whether patients and their families, physicians, health professionals or students of the area. The information in this site serves to enhance, not replace, the doctor-patient relationship.
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LAST UPDATE: July 25, 2010
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CAUTION Medical contents on the site are purely for guidance and information and cannot replace in any case the medical advice.
All contents provided by the site are written exclusively by professionals in the medical-scientific area, unless an explicit statement does not specify otherwise.- - Researchers demonstrate efficacy of antisense therapy for spinal muscular atrophy
- - Gene therapy rescues mice with SMA
- - Isis Pharmaceuticals towards an antisense drug
- - New finding clarifies the cause of SMA
- - Trans-splicing and gene therapy
- - Strengthening the junctions
- - New European funding for SMA research
- - New publications on SMA
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