用阿他芦醇治疗杜氏肌营养不良症。
Treatment with Ataluren for Duchene Muscular Dystrophy.
作者信息
Morkous Sameh S
机构信息
Department of Pediatrics, Pediatric Neurology Division, Lehigh Valley Children's Hospital, Allentown, PA.
出版信息
Pediatr Neurol Briefs. 2020 Dec 4;34:12. doi: 10.15844/pedneurbriefs-34-12.
Abstract
Investigators from Europe and the USA, representing the STRIDE Registry and Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), examined the effectiveness of ataluren and standard of care in the Registry versus stand of care alone in the CINRG DNHS.
摘要
来自欧洲和美国的研究人员，代表STRIDE注册库和国际神经肌肉研究合作组（CINRG）杜氏肌营养不良自然史研究（DNHS），比较了注册库中阿他芦醇与标准治疗联合使用的效果和CINRG DNHS中单纯标准治疗的效果。
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本文引用的文献
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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.依鲁替尼在 STRIDE 注册研究和 CINRG DMD 自然病史研究中的安全性和有效性比较。
J Comp Eff Res. 2020 Apr;9(5):341-360. doi: 10.2217/cer-2019-0171. Epub 2020 Jan 30.
2
Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry.依鲁替尼治疗具有无义突变杜氏肌营养不良症患者的使用情况：STRIDE 登记处的患者人口统计学和特征。
J Comp Eff Res. 2019 Oct;8(14):1187-1200. doi: 10.2217/cer-2019-0086. Epub 2019 Aug 15.
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Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy.阿他芦伦治疗杜氏肌营养不良症的临床潜力。
Degener Neurol Neuromuscul Dis. 2016 May 13;6:37-48. doi: 10.2147/DNND.S71808. eCollection 2016.
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Age at onset of first signs or symptoms predicts age at loss of ambulation in Duchenne and Becker Muscular Dystrophy: Data from the MD STARnet.杜氏和贝克型肌营养不良症首发体征或症状出现时的年龄可预测丧失行走能力的年龄：来自MD STARnet的数据。
J Pediatr Rehabil Med. 2016;9(1):5-11. doi: 10.3233/PRM-160361.
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