期刊
NEUROMUSCULAR DISORDERS
卷 16, 期 6, 页码 374-380出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.nmd.2006.03.005
关键词
spinal muscular atrophy; outcome measures; clinical trials; gross motor function measure; quantitative muscle testing
Spinal muscular atrophy is a genetic disease of the anterior horn cell with high morbidity rate in childhood. Certain drugs may be of benefit and are in or under consideration for Phase II trials. Outcome measures that are age appropriate and representative of disease activity remain under study. Several have not yet been validated for spinal muscular atrophy. The Gross Motor Function Measure(TM) is a measure of motor function. We showed previously that the Gross Motor Function Measure(TM) is a reliable outcome measure to assess motor function in children with spinal muscular atrophy. By collating our data from 40 spinal muscular atrophy patients, ages 5 through 17 years, we now show the validity of the Gross Motor Function Measure(TM) when compared to Quantitative Muscle Testing and ambulatory status in children with spinal muscular atrophy. The median for Gross Motor Function Measure(TM) total scores for walkers was 237 (range: 197-261) and for non-walkers, 64 (range: 4-177; P < 0.0001) with no distributional overlap. We conclude that the Gross Motor Function Measure(TM) is valid and sensitive as an outcome measure for clinical trials in pediatric spinal muscular atrophy. (C) 2006 Elsevier B.V. All rights reserved.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据