Novel approaches to treat muscular dystrophies

Muscular dystrophies (MD) are a clinically and generically heterogeneous gr oup of skeletal muscle-wasting diseases. Mutations in the dystrophin gene r esult in dystrophin deficiency, which constitutes the pathogenic basis of D uchenne and Becker MD (DMD and BMD). Several MD are caused by mutations in other recently identified genes coding for proteins linked to the sarcolemm a, the nuclear envelope or the contractile apparatus. In addition, several MD have been mapped to different chromosomal loci and for most of them, the identification of the molecular defect is underway. The immediate result i s an ongoing reclassification of the MD into disorders defined not by clini cal characteristics but specific genetic mutations. At present, therapy of Mn is based on symptomatic treatment and supportive care. Convincing eviden ce for clinical efficacy is only available for corticosteroids that also su ffer from frequent and severe side effects. Up to now, curative therapy is not available, although promising new molecular therapies are under investi gation in animal models of MD. Current treatment strategies are discussed a nd a perspective for effective molecular therapy is given.