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.
Abstract
摘要
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本文引用的文献
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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.
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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.
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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.
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[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.
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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.
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