在药物注册临床试验设置中将DMD基因变异类与运动结果的关联

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Yuan Fang1, Utkarsh J Dang2, Katherine I Illei3

  • 1Department of Pharmaceutical Sciences, Binghamton University School of Pharmacy and Pharmaceutical Sciences, NY.

Neurology. Genetics
|February 28, 2025
PubMed
概括

基因变异类别很少解释年轻杜申肌肉发育不良症 (DMD) 患者的运动功能变异性. 在DMD临床试验中,根据变异类型的子组分析可能对治疗反应没有信息.

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