Re: "Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy" by Wilson and Flotte.
作者信息
Shieh Perry B, Bönnemann Carsten G, Müller-Felber Wolfgang, Blaschek Astrid, Dowling James J, Kuntz Nancy L, Seferian Andreea M
机构信息
University of California at Los Angeles School of Medicine, Los Angeles, California, USA.
National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland, USA.
出版信息
Hum Gene Ther. 2020 Aug;31(15-16):787. doi: 10.1089/hum.2020.217.
Abstract
摘要
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本文引用的文献
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A multicenter, retrospective medical record review of X-linked myotubular myopathy: The recensus study.X 连锁肌小管肌病的多中心回顾性病历回顾：再统计研究。
Muscle Nerve. 2018 Apr;57(4):550-560. doi: 10.1002/mus.26018. Epub 2017 Dec 22.
2
A natural history study of X-linked myotubular myopathy.X连锁性肌管性肌病的自然史研究。
Neurology. 2017 Sep 26;89(13):1355-1364. doi: 10.1212/WNL.0000000000004415. Epub 2017 Aug 25.
3
Medical complications in long-term survivors with X-linked myotubular myopathy.X连锁性肌管性肌病长期存活者的医学并发症
J Pediatr. 1999 Feb;134(2):206-14. doi: 10.1016/s0022-3476(99)70417-8.
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