Gene therapy trial targets kids with spinal muscular atrophy who can sit but not stand
NCT ID NCT03381729
First seen Feb 01, 2026 · Last updated May 14, 2026 · Updated 15 times
Summary
This study tested a gene therapy called AVXS-101 in children aged 6 months to 5 years with spinal muscular atrophy (SMA) who could sit but not stand or walk. The goal was to see if the treatment was safe and could help them gain motor skills like standing or walking. The trial was stopped early, but results may still provide insights into treating SMA.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Johns Hopkins
Baltimore, Maryland, 21287, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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Stanford University
Stanford, California, 94305, United States
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UCLA
Los Angeles, California, 90095, United States
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UT Southwestern
Dallas, Texas, 75390, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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Washington University
St Louis, Missouri, 63130, United States
Conditions
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