One-Time gene infusion aims to save babies from fatal muscle disease
NCT ID NCT03837184
Summary
This study tested a single-dose gene therapy for babies under 6 months old with a severe, often fatal genetic muscle disease called spinal muscular atrophy type 1. The goal was to see if the one-time intravenous treatment could help these infants achieve a major milestone: sitting up on their own for at least 10 seconds by 18 months of age. The therapy works by delivering a functional copy of a missing gene directly to the body.
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