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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
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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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Regulatory
Neurology/psychiatric
FDA