J Korean Neurol Assoc.  2012 Nov;30(4):257-266.

Treatment of Duchenne Muscular Dystrophy: A Comprehensive Review

Affiliations
  • 1Department of Neurology, Yonsei University College of Medicine, Seoul, Korea. ycchoi@yuhs.ac

Abstract

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder due to the loss of dystrophin in muscle fiber. The deficiency of dystrophin produces severe progressive muscle degeneration which leads to progressive muscle weakness. Affected patients usually become unambulatory in their early teens, and suffer a respiratory failure before 20 years of age. In an attempt to improve quality of life and extend life span of DMD patients, various treatments have been challenged; corticosteroid trial, rehabilitation, cardiac and pulmonary managements, orthopedic interventions, and nutritional support. However, only corticosteroid therapy and non-invasive ventilation have shown a salutary effect on the clinical course of DMD. Recently, a better understanding of the DMD pathophysiology has provided the scientific basis for new treatment modalities including cell and molecular therapy. Although previous clinical trials have demonstrated the limitation and possibility of new therapies, antisense-mediated exon skipping technology is now emerging as a promising approach to restore dystrophin expression. This article summarizes the current challenges and recommendations of treatment approaches in DMD patients.

Keyword

Cell therapy; Corticosteroids; Duchenne muscular dystrophy; Gene therapy

MeSH Terms

Adolescent
Adrenal Cortex Hormones
Dystrophin
Exons
Genetic Therapy
Humans
Muscle Weakness
Muscles
Muscular Dystrophy, Duchenne
Noninvasive Ventilation
Nutritional Support
Orthopedics
Quality of Life
Respiratory Insufficiency
Tissue Therapy
Adrenal Cortex Hormones
Dystrophin
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