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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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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

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