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MDA 2023: Long-term motor gains seen for SMA children on Zolgensma

Children with spinal muscular atrophy (SMA) given Zolgensma (onasemnogene abeparvovec-xioi) gene therapy in clinical trials are maintaining, years later, the motor milestones they achieved in the original studies — and some have hit additional milestones even without further treatment. That’s according to data from long-term follow-up (LTFU)…