Sustainable efficacy of nusinersen in children with later-onset spinal muscular atrophy: a 3-year observational study.
作者信息
Feng Yi-Jie, Yu Yi-Cheng, Zhao Jing-Li, Xu Lu, Zhao Cong-Ying, Hua Yi, Sheng Guo-Xia, Chen Ting-Ting, Zhang Yi, Wei Lin-Lin, Gao Feng, Mao Shan-Shan
机构信息
Department of Neurology, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou 310052, China.
Department of Nephrology, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou 310052, China.
出版信息
World J Pediatr. 2025 Aug;21(8):846-851. doi: 10.1007/s12519-025-00938-y. Epub 2025 Jul 29.
Abstract
摘要
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本文引用的文献
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Improvements in Walking Distance during Nusinersen Treatment - A Prospective 3-year SMArtCARE Registry Study.依库珠单抗治疗后步行距离的改善 - 前瞻性 3 年 SMArtCARE 登记研究。
J Neuromuscul Dis. 2023;10(1):29-40. doi: 10.3233/JND-221600.
2
Nusinersen for spinal muscular atrophy types II and III: a retrospective single-center study in South Korea.依库珠单抗治疗脊髓性肌萎缩症 II 型和 III 型：韩国单中心回顾性研究。
World J Pediatr. 2023 May;19(5):450-459. doi: 10.1007/s12519-022-00638-x. Epub 2022 Nov 28.
3
Population-based assessment of nusinersen efficacy in children with spinal muscular atrophy: a 3-year follow-up study.基于人群的脊髓性肌萎缩症患儿中诺西那生钠疗效评估：一项3年随访研究
Brain Commun. 2022 Oct 31;4(6):fcac269. doi: 10.1093/braincomms/fcac269. eCollection 2022.
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Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study.在接受nusinersen 治疗的非瘫痪型 2 型和 3 型 SMA 患儿中上肢功能得到改善：前瞻性 3 年 SMArtCARE 登记研究。
Orphanet J Rare Dis. 2022 Oct 23;17(1):384. doi: 10.1186/s13023-022-02547-8.
5
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Eur J Paediatr Neurol. 2022 Jul;39:103-109. doi: 10.1016/j.ejpn.2022.06.001. Epub 2022 Jun 5.
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Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy.2 型和 3 型脊髓性肌萎缩症 24 个月的 nusinersen 疗效数据。
Ann Clin Transl Neurol. 2022 Mar;9(3):404-409. doi: 10.1002/acn3.51514. Epub 2022 Feb 15.
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Ann Clin Transl Neurol. 2021 Aug;8(8):1622-1634. doi: 10.1002/acn3.51411. Epub 2021 Jun 24.
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