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Pre-symptomatic risdiplam treatment for spinal muscular atrophy initiated at 12 days post-birth: 14-month safety and developmental outcomes.出生后12天开始的脊髓性肌萎缩症症状前利司扑兰治疗：14个月的安全性和发育结果。
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2
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Lancet Neurol. 2022 Dec;21(12):1110-1119. doi: 10.1016/S1474-4422(22)00339-8. Epub 2022 Oct 14.
3
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.每日一次利司扑兰治疗 2 型和非卧床 3 型脊髓性肌萎缩症（SUNFISH 研究 2 部分）的安全性和有效性：一项 3 期、双盲、随机、安慰剂对照试验。
Lancet Neurol. 2022 Jan;21(1):42-52. doi: 10.1016/S1474-4422(21)00367-7.
4
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J Neurol. 2023 May;270(5):2531-2546. doi: 10.1007/s00415-023-11560-1. Epub 2023 Feb 3.
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J Comp Eff Res. 2022 Apr;11(5):347-370. doi: 10.2217/cer-2021-0216. Epub 2022 Jan 18.
6
Safety of risdiplam in spinal muscular atrophy patients after short-term treatment with nusinersen.利司扑兰治疗脊髓性肌萎缩症患者短期应用依洛硫酸酯酶β后的安全性。
Muscle Nerve. 2024 Nov;70(5):1095-1098. doi: 10.1002/mus.28228. Epub 2024 Aug 13.
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Risdiplam in Type 1 Spinal Muscular Atrophy.利司扑兰治疗 1 型脊髓性肌萎缩症。
N Engl J Med. 2021 Mar 11;384(10):915-923. doi: 10.1056/NEJMoa2009965. Epub 2021 Feb 24.
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[Risdiplam for the treatment of spinal muscular atrophy].用于治疗脊髓性肌萎缩症的利司扑兰
Zh Nevrol Psikhiatr Im S S Korsakova. 2024;124(2):45-57. doi: 10.17116/jnevro202412402145.
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Long-Term Comparative Efficacy and Safety of Risdiplam and Nusinersen in Children with Type 1 Spinal Muscular Atrophy.利司扑兰与nusinersen 治疗 1 型脊髓性肌萎缩症儿童的长期疗效和安全性比较。
Adv Ther. 2024 Jun;41(6):2414-2434. doi: 10.1007/s12325-024-02845-6. Epub 2024 May 5.
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Risdiplam in Spinal Muscular Atrophy: Safety Profile and Use Through The Early Access to Medicine Scheme for the Paediatric Cohort in Great Britain.利司扑兰治疗脊髓性肌萎缩症：英国儿科队列通过早期药物获取计划的安全性概况及应用
J Neuromuscul Dis. 2024;11(2):361-368. doi: 10.3233/JND-230162.

本文引用的文献

1
Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study.onasemnogene abeparvovec基因疗法在D-A-CH地区脊髓性肌萎缩症儿童中的疗效和安全性：一项基于人群的观察性研究。
Lancet Reg Health Eur. 2024 Oct 7;47:101092. doi: 10.1016/j.lanepe.2024.101092. eCollection 2024 Dec.
2
The impact of three gene copies on clinical characteristics and effect of disease-modifying treatment in patients with spinal muscular atrophy: a systematic literature review.三个基因拷贝对脊髓性肌萎缩症患者临床特征及疾病修饰治疗效果的影响：一项系统文献综述
Front Neurol. 2024 Feb 29;15:1308296. doi: 10.3389/fneur.2024.1308296. eCollection 2024.
3
Safety Concerns with Nusinersen, Risdiplam, and Onasemnogene Abeparvovec in Spinal Muscular Atrophy: A Real-World Pharmacovigilance Study.脊髓性肌萎缩症中使用 nusinersen、risdiplam 和 onasemnogene abeparvovec 的安全性问题：一项真实世界的药物警戒研究。
Clin Drug Investig. 2023 Dec;43(12):949-962. doi: 10.1007/s40261-023-01320-4. Epub 2023 Nov 23.
4
[Expert consensus on newborn screening for spinal muscular atrophy (2023 edition)].脊髓性肌萎缩症新生儿筛查专家共识（2023年版）
Zhonghua Yi Xue Za Zhi. 2023 Jul 18;103(27):2075-2081. doi: 10.3760/cma.j.cn112137-20230310-00372.
5
Newborn screening for spinal muscular atrophy in Australia: a non-randomised cohort study.澳大利亚新生儿脊髓性肌萎缩症筛查：一项非随机队列研究。
Lancet Child Adolesc Health. 2023 Mar;7(3):159-170. doi: 10.1016/S2352-4642(22)00342-X. Epub 2023 Jan 17.

Pre-symptomatic risdiplam treatment for spinal muscular atrophy initiated at 12 days post-birth: 14-month safety and developmental outcomes.

作者信息

Sun Zhihao, Wu Liping, Hui Yufan, Zhong Yuhang, Yang Jiangtao, Sun Zhiyong, Huang Xiaoling, Xiong Hui

机构信息

Neonatal Disease Screening Center Dongguan Maternal and Children Health Hospital Dongguan China.

Dongguan Key Laboratory of Neonatal Genetic and Metabolic Disease Screening and Diagnosis and Treatment Dongguan China.

出版信息

Pediatr Investig. 2025 Apr 14;9(2):201-202. doi: 10.1002/ped4.70003. eCollection 2025 Jun.

DOI:10.1002/ped4.70003
PMID:40539005
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12175634/
Abstract
摘要