Treatment of muscular dystrophies.
1. Specific therapies to cure the muscular dystrophies are not yet available. Therapeutic trials designed on the basis of our understanding of the pathophysiology of these disorders have had only limited success. 2. However, recent investigations in Duchenne muscular dystrophy have identified the abnormal gene and the missing or defective gene product, dystrophin. 3. These discoveries provide information which will lead to more rational and specific therapeutic approaches. 4. The advances in genetic research have led to more effective preventive therapy. Gene mapping has been applied successfully in carrier detection and antenatal diagnosis, and specific gene probes will soon become available for carrier testing for the two most common forms of muscular dystrophy, Duchenne muscular dystrophy and myotonic dystrophy. 5. Supportive therapies for muscular dystrophy patients now include respiratory support for selected patients with chronic respiratory insufficiency. 6. This review will focus on the two most common muscular dystrophies, Duchenne muscular dystrophy and myotonic dystrophy.
Kingston
W J
WJ
Department of Neurology, University of Rochester Medical Center, NY 14642.
Moxley
R T
RT
D000818:Animals; D006801:Humans; D009136:Muscular Dystrophies / Q000188:drug therapy*; D009137:Muscular Dystrophy, Animal / Q000188:drug therapy*