Outlook for therapy in the muscular dystrophies.
Despite recent advances in our understanding of the muscular dystrophies, current therapy for these disorders remains chiefly supportive. With the exception of prednisone for Duchenne dystrophy, no effective treatment for these disorders has been found that will improve strength or even slow the progression of weakness. This review highlights current strategies for treating muscular dystrophies and discusses recent trials of various pharmacologic agents, including corticosteroids, anabolic steroids, growth factors and beta-adrenergic agonists. The outlook for effective gene therapy is also presented.
Tawil
R
R
Department of Neurology, University of Rochester, New York 14642, USA.
D000318:Adrenergic beta-Agonists / Q000627:therapeutic use; D045930:Anabolic Agents / Q000627:therapeutic use; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use; D015316:Genetic Therapy / Q000379:methods*; D019382:Human Growth Hormone / Q000627:therapeutic use; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000627:therapeutic use; D009136:Muscular Dystrophies / Q000188:drug therapy / Q000628:therapy*; D013256:Steroids
D000318:Adrenergic beta-Agonists; D045930:Anabolic Agents; D000893:Anti-Inflammatory Agents; D013256:Steroids; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I