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Autor:
Mariacristina Scoto, Clare Galtrey, Channa Hewamadduma, Paul Keane, James B Lilleker, Chiara Marini-Bettolo, Zoe Phillips, Becky Sharp, Andrew Wenham, Giovanni Baranello
Publikováno v:
Journal of Neurology, Neurosurgery & Psychiatry. 93:e2.147
BackgroundRisdiplam is the first oral spinal muscular atrophy (SMA) treatment and was made available in the UK through an Early Access to Medicines Scheme (EAMS), which enrolled patients between September 2020–July 2021.MethodsRisdiplam’s EAMS in