【杜氏肌营养不良的发病机制及治疗策略研究进展】。

Q3 Medicine

中国当代儿科杂志 Pub Date : 2025-07-15 DOI:10.7499/j.issn.1008-8830.2503105

Yi-Zhi Ye, Li-Wen Wu

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引用次数: 0

摘要

杜氏肌营养不良症（DMD）是一种x连锁隐性神经肌肉疾病，主要表现为骨骼肌进行性变性和坏死，由肌营养不良蛋白基因突变引起。DMD患者在儿童期通常表现为进行性肌肉无力和萎缩。目前，DMD的可用治疗方案仍然有限，其疗效也不理想。本文旨在系统综述DMD治疗策略的最新进展，包括分析各种治疗方法的机制、临床试验结果及其潜在的临床应用，以指导临床决策。

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[Research progress on the pathogenesis and treatment strategies of Duchenne muscular dystrophy].

Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder characterized primarily by progressive degeneration and necrosis of skeletal muscle, resulting from mutations in the Dystrophin gene. Patients with DMD typically present with progressive muscle weakness and atrophy during childhood. Currently, available treatment options for DMD remain limited and their efficacy is suboptimal. This review aims to provide a systematic overview of recent advances in therapeutic strategies for DMD, including an analysis of the mechanisms underlying various treatment approaches, outcomes from clinical trials, and their potential clinical applications, in order to inform and guide clinical decision-making.

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来源期刊

中国当代儿科杂志 Medicine-Pediatrics, Perinatology and Child Health

CiteScore

1.50

自引率

0.00%

发文量

5006

期刊介绍： The Chinese Journal of Contemporary Pediatrics (CJCP) is a peer-reviewed open access periodical in the field of pediatrics that is sponsored by the Central South University/Xiangya Hospital of Central South University and under the auspices of the Ministry of Education of China. It is cited as a source in the scientific and technological papers of Chinese journals, the Chinese Science Citation Database (CSCD), and is one of the core Chinese periodicals in the Peking University Library. CJCP has been indexed by MEDLINE/PubMed/PMC of the American National Library, American Chemical Abstracts (CA), Holland Medical Abstracts (EM), Western Pacific Region Index Medicus (WPRIM), Scopus and EBSCO. It is a monthly periodical published on the 15th of every month, and is distributed both at home and overseas. The Chinese series publication number is CN 43-1301/R；ISSN 1008-8830. The tenet of CJCP is to “reflect the latest advances and be open to the world”. The periodical reports the most recent advances in the contemporary pediatric field. The majority of the readership is pediatric doctors and researchers.