医学
脊髓性肌萎缩
形状记忆合金*
不利影响
肌营养不良
内科学
观察研究
评定量表
物理疗法
外科
疾病
心理学
发展心理学
数学
组合数学
作者
Maria Gavriilaki,Maria Moschou,Maria Pagiantza,Marianthi Arnaoutoglou,Vasilios Κ. Kimiskidis
摘要
Risdiplam was the first orally administered drug approved to treat spinal muscular atrophy (SMA). Efficacy in adults is based on short-term observational studies. This longitudinal study aimed to examine risdiplam's efficacy and safety in adults over a long period of follow-up.
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