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FDA Gives Broad Approval to Gene Therapy for Rare Neurologic Disease

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FDA Gives Broad Approval to Gene Therapy for Rare Neurologic Disease (MedPage Today) — The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in adults and pediatric patients 2 years and older with a confirmed mutation in the survival motor neuron…

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