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Duchenne muscular dystrophy (DMD) is a fatal, genetic disorder whose relentless progression underscores the urgency for developing a cure. Although Duchenne initiated clinical trials roughly 150 years ago, therapies for DMD remain supportive rather than curative. A paradigm shift towards developing rational therapeutic strategies occurred with identification of the DMD gene. Gene- and cell-based therapies designed to replace the missing gene and/or dystrophin protein have achieved varying degrees of success. However, pharmacological strategies not designed to replace dystrophin per se appear promising, and can circumvent many hurdles hampering gene- and cell-based therapy. Here, we will review present pharmacological strategies, in particular those dealing with functional substitution of dystrophin by utrophin and enhancing muscle progenitor commitment by myostatin blockade, with a view toward facilitating drug discovery for DMD.

Original publication




Journal article


Nat Rev Drug Discov

Publication Date





379 - 390


Animals, Calcium, Cytoskeletal Proteins, Disease Models, Animal, Drug Design, Drug Evaluation, Preclinical, Dystrophin, Homeostasis, Humans, Membrane Proteins, Muscular Dystrophies, Promoter Regions, Genetic, Utrophin