New therapies for Duchenne muscular dystrophy: challenges, prospects and clinical trials

Abstract

Muscular dystrophies primarily affect skeletal muscle. Mutations in a large number of genes, mainly encoding cytoskeletal proteins, cause different forms of dystrophy that compromise patient mobility and quality of life, and in the most severe cases lead to complete paralysis and premature death. Although muscular dystrophies still lack an effective therapy, several novel strategies are entering or are ready to enter clinical trials. Here we review the main experimental strategies, namely drug, gene and cell therapies, outlining their goals and limitations. We also provide an update of ongoing or planned clinical trials based on these strategies.