Duchenne Muscular Dystrophy: Contemporary Therapeutic Options and Real-World Challenges in Treatment Selection - PubMed
7 hours ago
- #Duchenne muscular dystrophy
- #gene therapy
- #treatment challenges
- DMD is a severe X-linked disorder caused by dystrophin mutations, leading to progressive muscle degeneration and motor decline.
- Diagnosis occurs in early childhood via motor delays, elevated creatine kinase, and genetic testing.
- Therapeutic options now include corticosteroids, exon-skipping therapies, nonsense mutation readthrough agents, and gene replacement strategies.
- No current therapy restores full dystrophin or completely halts disease progression, highlighting ongoing challenges.
- The review covers FDA-approved medications, mechanisms, efficacy, safety, and real-world treatment selection complexities.