Single infusion gene therapy offers hope for infants with devastating muscle disease

NCT ID NCT03837184

Completed Disease control Sponsor: Novartis Gene Therapies Source: ClinicalTrials.gov ↗

First seen Feb 01, 2026 · Last updated Apr 21, 2026 · Updated 15 times

Summary

This study tested a one-time gene replacement therapy for infants under 6 months old with spinal muscular atrophy type 1, a severe genetic muscle-wasting disease. Researchers gave a single intravenous infusion of the therapy called onasemnogene abeparvovec-xioi to see if it could help babies achieve important milestones like sitting alone. The main goal was to measure how many treated infants could sit unsupported for at least 10 seconds by 18 months of age.

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Contacts and locations

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Locations

National Taiwan University Hospital

Taipei, Taiwan
Pusan National University Yangsan Hospital

Yangsan, Gyeongsangnam-do, South Korea
Seoul National University Hospital

Seoul, South Korea
Tokyo Women's Medical University

Tokyo, Japan

Conditions

Explore the condition pages connected to this study.

SPINAL MUSCULAR ATROPHY TYPE I