医学
杜氏肌营养不良
临床试验
人口
物理医学与康复
重症监护医学
病理
内科学
环境卫生
标识
DOI:10.1016/j.ncl.2014.05.002
摘要
The dystrophinopathies Duchenne and Becker muscular dystrophies (DMD and BMD) represent the most common inherited disorders of muscle. Improvements in cardiac care, attention to respiratory function, and judicious use of spinal correction surgery have led to increased survival in the DMD population. Meanwhile, advances in molecular therapeutics have led to promising therapies that are in or are entering clinical trials. An understanding of the dystrophinopathies, and recent advances in their molecular diagnosis and treatment, is of benefit to practicing neurologists.
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