Long-Term Follow-up Study for Patients From AVXS-101-CL-101

Study Summary

This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.

Conditions

Spinal Muscular Atrophy 1

Interventions / Drugs

Onasemnogene Abeparvovec-xioi

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment13 participants

Primary Completion—

Lead SponsorNovartis Gene Therapies

Data Retrieved2026-04-11 02:55:49.152297+00:00

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