Muscular dystrophies: Mechanisms

Since the cloning of the gene responsible for Duchenne and Becker muscular dystrophy two decades ago, a number of other genes have been associated with various forms of muscular dystrophy. The protein products of these genes display a diversity of cellular localizations and functions, suggesting a complex pathophysiology within this class of disorders. Two recently identified functions of selected proteins include membrane repair and glycosylation, providing important insights into the disease process. A definitive cure remains elusive, but supportive therapies have become increasingly sophisticated, improving the quality of life and lengthening the life expectancy for many affected individuals.