Nusinersen in spinal muscular atrophy type 1 from neonates to young adult: 1-year data from three Asia-Pacific regions.
作者信息
Chan Sophelia Hoi-Shan, Chae Jong-Hee, Chien Yin-Hsiu, Ko Tae-Sung, Lee Jee Hun, Lee Yun Jeong, Nam Sang Ook, Jong Yuh-Jyh
机构信息
Department of Paediatrics and Adolescent Medicine, Queen Mary Hospital, The University of Hong Kong, Hong Kong SAR, China
Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine, Seoul, The Republic of Korea.
出版信息
J Neurol Neurosurg Psychiatry. 2021 Nov;92(11):1244-1246. doi: 10.1136/jnnp-2020-324532. Epub 2021 Feb 11.
Abstract
摘要
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本文引用的文献
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Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study.在脊髓性肌萎缩症的前症状期对婴儿期开始用 nusinersen：2 期 NURTURE 研究的中期疗效和安全性结果。
Neuromuscul Disord. 2019 Nov;29(11):842-856. doi: 10.1016/j.nmd.2019.09.007. Epub 2019 Sep 12.
2
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy.SMA 类型 1 婴儿接受单次基因替换治疗的 1/2A 期研究中年龄和运动功能的影响。
Pediatr Neurol. 2019 Sep;98:39-45. doi: 10.1016/j.pediatrneurol.2019.05.005. Epub 2019 May 13.
3
Natural history of infantile-onset spinal muscular atrophy.婴儿型脊髓性肌萎缩症的自然病史。
Ann Neurol. 2017 Dec;82(6):883-891. doi: 10.1002/ana.25101. Epub 2017 Dec 8.
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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.依库珠单抗治疗婴儿型脊髓性肌萎缩症的疗效观察
N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752.
5
Presymptomatic Diagnosis of Spinal Muscular Atrophy Through Newborn Screening.通过新生儿筛查对脊髓性肌萎缩症进行症状前诊断。
J Pediatr. 2017 Nov;190:124-129.e1. doi: 10.1016/j.jpeds.2017.06.042. Epub 2017 Jul 12.
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