Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls.
Basil T DarrasRiccardo MassonMaria Mazurkiewicz-BełdzińskaKristy RoseHui XiongEdmar ZanoteliGiovanni BaranelloClaudio BrunoDmitry VlodavetsYi WangMuna El-KhairiMarianne GerberKsenija GorniOmar KhwajaHeidemarie KletzlRenata S ScalcoPaulo FontouraLaurent Servaisnull nullPublished in: The New England journal of medicine (2021)
In this study involving infants with type 1 SMA, risdiplam resulted in higher percentages of infants who met motor milestones and who showed improvements in motor function than the percentages observed in historical cohorts. Longer and larger trials are required to determine the long-term safety and efficacy of risdiplam in infants with type 1 SMA. (Funded by F. Hoffmann-La Roche; FIREFISH ClinicalTrials.gov number, NCT02913482.).
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