Duchenne muscular dystrophy: pathogenesis and pharmacology

Abstract Duchenne muscular dystrophy (DMD) is an inherited progressive disease in which segmental necrosis of muscle fibres leads to skeletal muscle degeneration. At present, no cure is available and the patients' management is only supportive. As the altered gene product is not yet known, most clinical trials of therapeutic agents have been aimed at arresting or slowing down the necrotic process or promoting the regeneration of muscle. When more is known about the altered gene product (presumably a protein) and how it produces DMD, it may be possible to design drugs for specific treatment of the disease. At present, proposed treatments range from preventing calcium accumulation to inhibiting hormone release. In this review, J. Elbrink, S. K. Malhotra and M.G. Elleker outline the various approaches to treatment in the past and consider avenues that could be promising in the future .