FDA Approves Innovative Gene Therapy to Treat Pediatric Patients with Spinal Muscular Atrophy

The U.S. Food and Drug Administration approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality.

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