One-Time gene therapy helps babies with rare muscle disease sit on their own
NCT ID NCT03837184
First seen Feb 01, 2026 · Last updated May 24, 2026 · Updated 17 times
Summary
This study tested a one-time gene therapy (AVXS-101) in infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The main goal was to see if treated babies could sit without support for at least 10 seconds by 18 months of age. The therapy aims to replace the missing SMN1 gene to improve muscle function and survival.
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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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National Taiwan University Hospital
Taipei, Taiwan
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Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, South Korea
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Seoul National University Hospital
Seoul, South Korea
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Tokyo Women's Medical University
Tokyo, Japan
Conditions
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