医学
杜氏肌营养不良
肌营养不良
肌营养不良蛋白
弱点
肌肉无力
生活质量(医疗保健)
重症监护医学
物理疗法
生物信息学
物理医学与康复
儿科
内科学
外科
护理部
生物
作者
Eszter Czifrus,Daniel J. Berlau
标识
DOI:10.1080/14740338.2024.2394578
摘要
Introduction Duchenne muscular dystrophy (DMD) is a X-linked genetic disorder characterized by progressive muscle degeneration and weakness, caused by mutations in the dystrophin gene. DMD has effects in early age with significantly shortened lifespan and deteriorated quality of life in the second decade creating an urgent need to develop better therapeutic options. Corticosteroid medication therapy is an integral tool for the management of DMD and several therapeutic options have been recently approved for use.
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