25-11-2025 18:34 via medpagetoday.com

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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