AVXS-101 Phase 1 Gene Therapy Clinical Trial in Spinal Muscular Atrophy Type 1 (SMA1): Event-free Survival and Achievement of Developmental Milestones

被引：0
|
作者：
Mendell, J.
Al-Zaidy, S.
Shell, R.
Arnold, W.
Rodino-Klapac, L.
Prior, T.
Lowes, L.
Alfano, L.
Berry, K.
Church, K.
Kissel, J.
Nagendran, S.
L'Italien, J.
Sproule, D.
Wells, C.
机构：
来源：
ANNALS OF NEUROLOGY | 2018年 / 84卷
关键词：
Neuromuscular Disorders; Genetics; Rare Diseases;
D O I：
暂无
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
136
引用
收藏
页码：S387 / S388
页数：2
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