Yuewei Chi

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Safety and Efficacy of Nusinersen and Risdiplam for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials

Oct 6, 2023 | mdpi.com | Yue Qiao |Yuewei Chi |Jian Gu |Ying Ma

1. IntroductionSpinal muscular atrophy (SMA) is a debilitating hereditary neuromuscular disorder characterized by progressive muscle weakness and atrophy, hypotonia, and respiratory distress [1]. The clinical features of SMA vary in degrees of severity but all patients have in common progressive muscle weakness and mobility impairment [2].

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