《杜氏肌营养不良症的肺部终点：研讨会总结》。
Pulmonary Endpoints in Duchenne Muscular Dystrophy. A Workshop Summary.
机构信息
1 Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania.
2 Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania.
出版信息
Am J Respir Crit Care Med. 2017 Aug 15;196(4):512-519. doi: 10.1164/rccm.201703-0507WS.
Development of novel therapeutics for treatment of Duchenne muscular dystrophy (DMD) has led to clinical trials that include pulmonary endpoints that allow assessment of respiratory muscle status, especially in nonambulatory subjects. Parent Project Muscular Dystrophy (PPMD) convened a workshop in Bethesda, Maryland, on April 14 and 15, 2016, to summarize published respiratory data in DMD and give guidance to clinical researchers assessing the effect of interventions on pulmonary outcomes in DMD.
新型治疗药物的开发用于治疗杜氏肌营养不良症（DMD），已经开展了临床试验，包括肺部终点，以评估呼吸肌状况，特别是在非运动 subjects.Parent 项目肌肉营养不良症（PPMD）在马里兰州贝塞斯达召开了一个研讨会，2016 年 4 月 14 日和 15 日，总结了 DMD 的已发表的呼吸数据，并指导临床研究人员评估干预对 DMD 肺部结果的影响。
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