This Phase 3 study is a long-term follow-up of safety and efficacy for people with spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi (Zolgensma) in earlier trials. Participants roll over from their parent study into this study for continued monitoring of safety and of how well and how long the treatment effect lasts. Only patients who received the gene replacement therapy in a Novartis Gene Therapies-sponsored study, with consent from the participant or legal guardian, may take part.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study * Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule Exclusion Criteria: * Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study