ASPIRO Gene therapy trial in X-linked myotubular myopathy (XLMTM): Update on preliminary safety and efficacy findings up to 72 weeks post-treatment

被引：0

|

作者：

Shieh, P. ^{[1
]}

Kuntz, N. ^{[2
]}

Bonnemann, C. ^{[3
]}

Mueller-Felber, W. ^{[4
]}

Lawlor, M. W. ^{[5
]}

Servais, L. ^{[6
]}

Smith, B. ^{[7
]}

Noursalehi, M. ^{[8
]}

Prasad, S. ^{[8
]}

Dowling, J. ^{[9
]}

机构：

[1] Univ Calif Los Angeles, Los Angeles, CA 90024 USA

[2] Childrens Hosp, Chicago, IL USA

[3] NIH, Bldg 10, Bethesda, MD 20892 USA

[4] Klinikum Univ, Munich, Germany

[5] Med Coll Wisconsin, Milwaukee, WI 53226 USA

[6] Hop Armand Trousseau, Paris, France

[7] Univ Florida, Gainesville, FL USA

[8] Audentes Therapeut, San Francisco, CA USA

[9] Hosp Sick Children, Toronto, ON, Canada

来源：

NEUROMUSCULAR DISORDERS | 2020年 / 30卷

关键词：

D O I：

10.1016/j.nmd.2020.08.070

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

P.62

引用

收藏

页码：S65 / S66

页数：2

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