One-Time gene therapy helps babies with rare muscle disease sit on their own
NCT ID NCT03837184
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a one-time gene replacement therapy 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, but ongoing management is still needed.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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
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