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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