Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial

被引：0
|
作者：
Mendell, J. [1 ]
Shell, R. [1 ]
Lehman, K. [1 ]
McColly, M. [1 ]
Lowes, L. [1 ]
Alfano, L. [1 ]
Miller, N. [1 ]
Iammarino, M. [1 ]
Church, K. [1 ]
Ogrinc, F. [2 ]
Ouyang, H. [2 ]
Kernbauer, E. [2 ]
Shah, S. [2 ]
L'Italien, J. [2 ]
Sproule, D. [2 ]
Feltner, D. [2 ]
Al-Zaidy, S. [1 ]
机构：
[1] Nationwide Childrens Hosp, Columbus, OH USA
[2] AveXis Inc, Bannockburn, IL USA
来源：
NEUROMUSCULAR DISORDERS | 2019年 / 29卷
关键词：
D O I：
10.1016/j.nmd.2019.06.513
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
P.351
引用
收藏
页码：S184 / S184
页数：1
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