Spinal Muscular Atrophy Type I research hub

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

This study is checking on people with Spinal Muscular Atrophy (SMA) who have already had a special gene treatment called onasemnogene abeparvovec-xioi. We want to see how they are doing in the long term, both with their health and how well the treatment keeps working.