Gene therapy breakthrough: one dose may help babies with rare muscle disease
NCT ID NCT03505099
First seen Feb 01, 2026 · Last updated May 22, 2026 · Updated 15 times
Summary
This study tested a single dose of a gene therapy called AVXS-101 in 30 infants under 6 weeks old who were genetically diagnosed with spinal muscular atrophy (SMA) but not yet showing symptoms. The goal was to see if the treatment could help them reach motor milestones like sitting or standing alone. While the therapy aims to control the disease, ongoing management may still be needed.
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This is a summary of
the original study
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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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Canada Childrens Hospital of Eastern Ontario
Ottawa, Ontario, K1H8L1, Canada
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Centre Hospitalier Régional Hôpital La Citadelle
Liège, 4000, Belgium
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Medical Center Dallas
Dallas, Texas, 75235, United States
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Clinic for Special Children
Strasburg, Pennsylvania, 17579, United States
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Columbia University Medical Center
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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Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH, United Kingdom
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Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, 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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St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
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Sydney Children's Hospital
Randwick, New South Wales, 2145, Australia
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Tokyo Women's Medical
Tokyo, Japan
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University Hospital and UW Health Clinics
Madison, Wisconsin, 53792, United States
Conditions
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