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Nusinersen improves motor function in children with later-onset spinal muscular atrophy: The CHERISH trial

01:24 EST 15 Feb 2018 | 2 Minute Medicine

1. Intrathecal injections of nusinersen in pediatric later-onset spinal muscular atrophy (SMA) patients were associated with increased clinical motor function scores compared to sham treated patients. 2. The overall safety profile of nusinersen in this trial was comparable to sham treated patients. Evidence Rating Level: 1 (Excellent) Study Rundown: SMA is a rare and debilitating […]

Original Article: Nusinersen improves motor function in children with later-onset spinal muscular atrophy: The CHERISH trial

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