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首页> Muscle & nerve> Longitudinal Efficacy of Nusinersen Treatment on Health-Related Quality of Life and Independence in Children With Later-Onset Spinal Muscular Atrophy.

Longitudinal Efficacy of Nusinersen Treatment on Health-Related Quality of Life and Independence in Children With Later-Onset Spinal Muscular Atrophy.

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作者： Siyi,Huang ^[1] ; Liya,Jiang ^[1] ; Dongming,Zhou ^[2] ; Yue,Yan ^[1] ; Yijie,Feng ^[1] ; Yicheng,Yu ^[1] ; Mei,Yao ^[3] ; Feng,Gao ^[1] ; Shanshan,Mao ^[1]

作者单位： Department of Neurology, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China. ^[1] Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China. ^[2] Department of Infectious Diseases, Children's Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China. ^[3]

关键词 SMAIS‐ULM nusinersen patient‐reported outcome quality of life spinal muscular atrophy

主题词人类(Humans);生活质量(Quality of Life);男(雄)性(Male);女(雌)性(Female);寡核苷酸类(Oligonucleotides);儿童(Child);儿童, 学龄前(Child, Preschool);纵向研究(Longitudinal Studies);治疗结果(Treatment Outcome);肌萎缩, 脊髓性(Muscular Atrophy, Spinal);青少年(Adolescent);婴儿(Infant);日常生活活动(Activities of Daily Living);医疗护理提供者(Caregivers);儿童脊髓性肌萎缩(Spinal Muscular Atrophies of Childhood)

DOI 10.1002/mus.28311

PMID 39718202

发布时间 2025-02-08

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Muscle & nerve

Muscle & nerve

2025年71卷3期

368-376页

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