Outlook for Therapy in the Muscular Dystrophies

ABSTRACT

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 β-adrenergic agonists. The outlook for effective gene therapy is also presented.

Keywords

Muscular dystrophy - prednisone - steroid therapy - growth factors - β-adrenergic agonists - gene therapy