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Search results for "Spinal muscular atrophy"

The FDA ( U.S. Food and Drug Administration ) has approved Spinraza ( Nusinersen ), the first drug approved to treat children and adults with spinal muscular atrophy ( SMA ), a rare and often fatal ge ...


The U.S. Food and Drug Administration ( FDA ) has approved Zolgensma ( Onasemnogene abeparvovec-xioi ), the first gene therapy approved to treat children less than two years of age with spinal muscula ...