杜氏肌营养不良症生物学治疗的研究进展  

Duchenne muscular dystrophy and its treatment options: A brief review

作　　者：刘悦 马沛沛 吴士文[1] LIU Yue;MA Peipei;WU Shiwen(Department of Neurology,General Hospital of Chinese People's Armed Police Force,Beijing 100039,China)

机构地区：[1]武警总医院神经内科,北京100039

出　　处：《中华灾害救援医学》2018年第4期233-236,共4页Chinese Journal of Disaster Medicine

基　　金：首都临床特色应用研究与成果推广项目(Z151100004015025);北京市科技计划课题(Z151100003915126)

摘　　要：杜氏肌营养不良症(Duchenne muscular dystrophy,DMD)是由于dystrophy基因突变所致的一种致死性X连锁隐性遗传病。随着糖皮质激素(glucocorticoids,GCs)的广泛应用,多学科综合护理水平的提高,心脏和呼吸系统并发症的处理,DMD患者的生存质量得到明显提高,生存时间亦得以延长。近年来,随着对包括产前治疗性干预在内的一系列治疗途径的研究及临床应用,进一步降低了患者的发病率和病死率。本文对DMD生物学治疗的研究现状进行综述,并分析不同治疗策略在其治疗中的研究进展和潜力。Duchenne muscular dystrophy(DMD)is a fatal X-linked recessive hereditary disease caused by mutation of dystrophy gene.The life quality of DMD patients has been significantly improved,and the survival time prolonged because of the wide application of glucocorticoids(GCs)and the improvement of multidisciplinary comprehensive nursing and the treatment of the complications of heart and respiratory system.In recent years,a series of therapeutic approaches such as prenatal therapeutic intervention have further reduced the incidence and fatality rate of the patients.In this paper,the research status of DMD therapy is reviewed,and the research progress and potential of different treatment options are also analyzed.

关 键 词：杜氏肌营养不良症 生物学治疗 抗肌萎缩蛋白 

分 类 号：R746.2[医药卫生—神经病学与精神病学]