Clinical analysis of nusinersen for spinal muscular atrophy types 2 and 3 in children (PubMed, Zhongguo Dang Dai Er Ke Za Zhi) - Sep 18, 2026 - "Nusinersen is well tolerated and safe in children with SMA types 2 and 3. The proportions achieving clinically meaningful responses on motor-function scales and CMAP improvements increase over time, and rehabilitation training may enhance efficacy."
Journal • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases
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Ci-Liu Zhang; Fang-Yun Liu; Guo-Li Wang; Hao-Lin Duan; Jing Peng; Tian-Ming Wang; Yi-Wen Xu
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