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BioWorld - Wednesday, March 4, 2026
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» PTC’s risdiplam gets FDA nod as first at-home SMA therapy
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PTC’s risdiplam gets FDA nod as first at-home SMA therapy
Aug. 7, 2020
By
Jennifer Boggs
More than two weeks ahead of its expected PDUFA date, PTC Therapeutics Inc.’s spinal muscular atrophy (SM) drug, risdiplam, gained FDA approval, making it the first at-home, oral treatment intended for use in adults and children 2 months and older.
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Neurology/psychiatric
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