ASPIRO phase 1/2 gene therapy trial in X-linked motubular myopathy (XLMTM): update on preliminary safety and efficacy findings

被引：1
|
作者：
Dowling, J. [1 ]
Shieh, P. [2 ]
Kuntz, N. [3 ]
Bonnemann, C. [4 ]
Muller-Felber, W. [5 ]
Lawlor, M. [6 ]
Servais, L. [7 ]
Smith, B. [8 ]
Noursalehi, M. [9 ]
Rico, S. [9 ]
Prasad, S. [9 ]
机构：
[1] Hosp Sick Children, Toronto, ON M5G 1X8, Canada
[2] Univ Calif Los Angeles, Los Angeles, CA USA
[3] Childrens Hosp Chicago, Chicago, IL USA
[4] NIH, Bldg 10, Bethesda, MD 20892 USA
[5] Univ Munich, Munich, Germany
[6] Med Coll Wisconsin, Milwaukee, WI 53226 USA
[7] Hop Armand Trousseau, Paris, France
[8] Univ Florida, Gainesville, FL USA
[9] Audentes Therapeut, San Francisco, CA USA
来源：
NEUROMUSCULAR DISORDERS | 2019年 / 29卷
关键词：
D O I：
10.1016/j.nmd.2019.06.593
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
O.39
引用
收藏
页码：S207 / S207
页数：1
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