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Medical News & Perspectives
September 26, 2007

Studies Point Way to New Therapeutic Prospects for Muscular Dystrophy

JAMA. 2007;298(12):1385-1386. doi:10.1001/jama.298.12.1385

Although 20 years have elapsed since scientists identified the protein encoded by the defective gene that causes Duchenne muscular dystrophy (DMD), treatment options to help slow the disease's progressive muscle degeneration have remained limited.

In recent years, however, therapeutic prospects have become brighter. Scientists focusing on the disease have developed some promising potential treatments, some of which are in clinical trials. In addition, a few researchers from outside the field have made some fortuitous discoveries about muscle maintenance and regeneration that are providing insights that may one day lead to new treatments.