Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Study Summary

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.

Conditions

Spinal Muscular Atrophy Type I Spinal Muscular Atrophy Type II Spinal Muscular Atrophy Type III SMA

Interventions / Drugs

Onasemnogene Abeparvovec-xioi

View on ClinicalTrials.gov ↗

Sponsor

Study Details

Enrollment85 participants

Primary Completion—

Lead SponsorNovartis Gene Therapies

Data Retrieved2026-04-11 02:55:49.152655+00:00

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