Research progress on the pathogenesis and treatment strategies of Duchenne muscular dystrophy.
10.7499/j.issn.1008-8830.2503105
- Author:
Yi-Zhi YE
1
;
Li-Wen WU
Author Information
1. Department of Neurology, Hunan Children's Hospital, Changsha 410007, China.
- Publication Type:English Abstract
- Keywords:
Duchenne muscular dystrophy;
Gene therapy;
Pathogenesis;
Pharmacological treatment
- MeSH:
Muscular Dystrophy, Duchenne/genetics*;
Humans;
Genetic Therapy
- From:
Chinese Journal of Contemporary Pediatrics
2025;27(9):1143-1148
- CountryChina
- Language:Chinese
-
Abstract:
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.
