Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.
Thomas O CrawfordBasil T DarrasJohn W DaySally Dunaway YoungTina DuongLeslie L NelsonDoreen BarrettGuochen SongSanela BilicShaun M CoteMara SadanowiczRyan IarrobinoTiina J XuJanet O'NeilJosé RosselloAmy PlaceNathalie KerteszGeorge NomikosYung ChyungPublished in: Neurology (2024)
This study provides Class III evidence that apitegromab improves motor function in later-onset types 2 and 3 spinal muscular atrophy.
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