209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7-9 November 2014, Heemskerk, The Netherlands

被引：163

作者：

Finkel, Richard ^{[1
]}

Bertini, Enrico ^{[2
]}

Muntoni, Francesco ^{[3
]}

Mercuri, Eugenio ^{[4
]}

机构：

[1] Nemours Childrens Hosp, Orlando, FL 32827 USA

[2] UCL Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

[3] Bambino Gesu Childrens Res Hosp, Rome, Italy

[4] Univ Cattolica Sacro Cuore, Pediat Neurol Unit, I-00168 Rome, Italy

来源：

NEUROMUSCULAR DISORDERS | 2015年 / 25卷 / 07期

关键词：

FUNCTIONAL MOTOR SCALE; PLACEBO-CONTROLLED TRIAL; SMN2 COPY NUMBER; DOUBLE-BLIND; MOUSE MODEL; YOUNG-CHILDREN; WALK TEST; SMA; RELIABILITY; VALIDATION;

D O I：

10.1016/j.nmd.2015.04.009

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

[No abstract available]

引用

页码：593 / 602

页数：10

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