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Genetic and Rare Diseases Information Center (GARD)

Spinal muscular atrophy


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How might spinal muscular atrophy be treated?

There is currently no specific cure for spinal muscular atrophy. Infants who have a severe form of the disease frequently die of respiratory failure due to weakness of the muscles that help with breathing. Children who have milder forms will live much longer but may need extensive medical support.[1]

The current treatment for spinal muscular atrophy involves prevention and management of the secondary effects of muscle weakness and loss. Respiratory, nutritional and rehabilitation care are available. In addition, several drugs have been identified in laboratory experiments that may help some patients. Some of the drugs that are currently being investigated include: Butyrates, valproic acid, hydroxyurea, and riluzole.[1]
Last updated: 11/28/2011

  1. Learning About Spinal Muscular Atrophy. National Human Genome Research Institute (NHGRI). October 19, 2009; Accessed 4/21/2010.

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Management Guidelines

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Clinical Trials & Research for this Disease

  • lists trials that are studying or have studied Spinal muscular atrophy. Click on the link to go to to read descriptions of these studies.
  • The Research Portfolio Online Reporting Tool (RePORT) provides access to reports, data, and analyses of research activities at the National Institutes of Health (NIH), including information on NIH expenditures and the results of NIH-supported research. Although these projects may not conduct studies on humans, you may want to contact the investigators to learn more. To search for studies, enter the disease name in the "Text Search" box. Then click "Submit Query".
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