New therapies in muscular dystrophies.
Muscular dystrophies are a group of hereditary muscle disorders that often result in severe disability. Curative therapy is not yet available for muscular dystrophies (MD). In the near future, it is not expected that gene-replacement therapy will be available. Other strategies to decrease the rate of muscle necrosis and to increase strength in patients are necessary. Therefore the interest in symptomatic drug treatment has recently increased. A few trials have been performed on different types of muscular dystrophies, and some have generated positive results on muscle strength or muscle mass. We review the state of the art in therapy of MD and summarize the drugs that have been used and the evidence and results of such clinical trials.
Angelini
C
C
Department of Neurology, University of Padua, Via Giustiniani 5, I-35128 Padua, Italy.
Bonifati
D M
DM
D002986:Clinical Trials as Topic; D015316:Genetic Therapy; D006801:Humans; D009136:Muscular Dystrophies / Q000188:drug therapy* / Q000378:metabolism / Q000503:physiopathology; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy / Q000378:metabolism / Q000503:physiopathology; D020391:Muscular Dystrophy, Facioscapulohumeral / Q000188:drug therapy / Q000378:metabolism / Q000503:physiopathology