Can gene therapy zolgensma safely improve motor skills in SMA?

This study follows 80 people with spinal muscular atrophy (SMA) in Japan who receive Zolgensma (onasemnogene abeparvovec) as an intrathecal injection. The goal is to track safety issues, such as liver problems or blood clotting abnormalities, and to measure changes in motor function using a standard SMA assessment scale. Since it's an observational study, patients receive standard care, and researchers simply collect data from their doctors. The findings could help clarify how well this gene therapy works in real-world clinical practice.