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Updated: Jul 27, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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用nusinersen治疗的SMA儿童的呼吸和睡眠结果 - 现实世界经验

K Gonski1, S Chuang2, A Teng3

  • 1Department of Respiratory Medicine Departments, Sydney Children's Hospital, Randwick, Australia; Department of Sleep Medicine, Sydney Children's Hospital, Randwick, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Sydney, Randwick, Australia.

Neuromuscular disorders : NMD
|June 8, 2023
PubMed
概括

努辛森治疗改善了脊髓肌肉缩 (SMA) 的儿童在睡眠期间的氧气水平. 虽然一些患者停止了夜间非侵入性通风 (NIV),但肺功能没有显著改善,显示呼吸系统结果稳定.

关键词:
孩子 孩子 孩子 孩子这就是Nusinersen.儿科 儿科 儿科多重睡眠学术 (Polysomnography) 是一种多重睡眠学术.脊柱肌肉缩 脊柱肌肉缩 脊柱肌肉缩螺旋测量是一种螺旋测量.

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