Treatment Advances in Spinal Muscular Atrophy

被引：0
|
作者：
Diana Bharucha-Goebel
Petra Kaufmann
机构：
[1] Children’s National Medical Center,Department of Neurology
[2] Neuromuscular and Neurogenetic Disorders of Childhoood Section (NNDCS)/NINDS/NIH,undefined
[3] National Center for Advancing Translational Sciences (NCATS)/NIH,undefined
来源：
Current Neurology and Neuroscience Reports | 2017年 / 17卷
关键词：
Spinal muscular atrophy; Clinical trials; Gene therapy; Natural history; Functional biomarkers; Newborn screening; Standard of care;
D O I：
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学科分类号：
摘要：
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