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Duchenne 근디스트로피의 치료에 대한 고찰

Other Titles
 Treatment of Duchenne Muscular Dystrophy: A Comprehensive Review 
Authors
 박형준 ; 최영철 
Citation
 대한신경과학회지, Vol.30(4) : 257~266, 2012 
Journal Title
 대한신경과학회지 
ISSN
 1225-7044 
Issue Date
2012
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.
URI
http://ir.ymlib.yonsei.ac.kr/handle/22282913/92271
Appears in Collections:
1. 연구논문 > 1. College of Medicine > Dept. of Neurology
Yonsei Authors
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