Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study.
Gyula AcsadiThomas O CrawfordWolfgang Müller-FelberPerry B ShiehRandal RichardsonNiranjana NatarajanDiana CastroDaniela Ramirez-SchremppGiulia GambinoPeng SunWildon FarwellPublished in: Muscle & nerve (2021)
Nusinersen demonstrated a favorable long-term benefit-risk profile in this broad population of individuals with infantile- or later-onset SMA.
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