One-Time gene therapy helps babies with fatal muscle disease sit and survive
NCT ID NCT03306277
First seen Jan 28, 2026 · Last updated Apr 30, 2026 · Updated 15 times
Summary
This study tested a one-time gene therapy (AVXS-101) in 22 infants under 6 months old with spinal muscular atrophy type 1, a severe muscle-weakening disease. The goal was to see if the treatment helped babies sit independently for at least 30 seconds and survive without needing permanent breathing support. Results showed significant improvements in both sitting ability and survival compared to natural history.
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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 and 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 Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Columbia University
New York, New York, 10032, United States
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David Geffen School of Medicine at UCLA
Los Angeles, California, 90095, United States
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Duke University
Durham, North Carolina, 27713, United States
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Johns Hopkins Pediatric Neurology
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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Oregon Health and Science University
Portland, Oregon, 97239, United States
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Stanford University
Stanford, California, 94305, United States
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University of Texas Southwestern Medical Center
Dallas, Texas, 75235, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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University of Wisconsin (Madison)
Madison, Wisconsin, 53792, United States
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Washington Unviersity School of Medicine
St Louis, Missouri, 63110, United States
Conditions
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