Duchenne muscular dystrophy (DMD) is an X-linked muscle wasting disease caused by the absence of the 427-kDa cytoskeletal protein, dystrophin. There is currently no effective therapy. Recent advances in the understanding of the disease are leading to many different approaches to treatment which enhance the physiological performance of the muscle. Delivery of the dystrophin gene or minigenes using plasmids or viruses, exon skipping of mutations induced by oligonucleotides and readthrough of nonsense mutations are all moving into the clinic. Several pharmacological approaches which improve the function of muscles in patients are also being tested and upregulation of other proteins has been shown to prevent the dystrophic process. Muscle can be repopulated with myoblasts or stem cells. All, or a combination, of these approaches hold great promise for the treatment of this devastating disease in the next decade.