CLINICAL TRIAL READINESS IN NON-AMBULATORY BOYS AND MEN WITH DUCHENNE MUSCULAR DYSTROPHY: MDA-DMD NETWORK FOLLOW-UP
CLINICAL TRIAL READINESS IN NON-AMBULATORY BOYS AND MEN WITH DUCHENNE MUSCULAR DYSTROPHY: MDA-DMD NETWORK FOLLOW-UP
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DOI：
10.1002/mus.25089
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发表时间：
2016-10-01
期刊：
影响因子：
3.4
通讯作者：
Miller, J. Philip
中科院分区：
文献类型：
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作者：
Connolly, Anne M.;Florence, Julaine M.;Miller, J. Philip
简介：患有杜氏肌营养不良症 (DMD) 的不能行走的男孩/男性的结果对随时间变化的敏感性尚不明确。方法：对受试者（n = 91；16.8 +/- 4.5 岁）进行为期 2 年的基线评估和每 6 个月一次的评估。我们使用意向治疗模型分析了所有受试者以及具有布鲁克量表评分的较强受试者的子集
Introduction: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. Methods: Subjects (n = 91; 16.8 +/- 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score