Historically, most boys who have Duchenne do not survive beyond their late twenties. Most commonly, diagnosis happens between 3-6 years old, ambulation is lost between the ages of 10-12, and breathing and feeding devices are needed in the late teens to early 20's. The simplest of tasks (like dressing, washing/brushing hair, brushing teeth, etc.) become difficult and in the later stages, heart and breathing muscles begin to fail. Nearly 15,000 boys are living with the disease in the United States alone and over 300,000 worldwide. The disorder knows no cultural, economic, or social boundaries.

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Muscular dystrophies are a group of genetic disorders characterized by muscle weakness. Duchenne is the most common and severe form, caused by loss of dystrophin, beneath the sarcolemma. The molecular mechanisms of the disease have been extensively investigated since the discovery of the gene in 1986.

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Initial signs of cardiac dysfunction caused by Duchenne muscular dystrophy are usually detected during adolescence. However, decreased physical activity, even if wheelchair-bound, can allow better tolerance of decreased cardiac function.

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The good news is that new gene-based therapies have recently emerged with noted advances in using conventional gene replacement strategies, RNA-based technology, and pharmacological approaches. In particular, antisense-mediated exon skipping has shown encouraging results and holds promise for the treatment of dystrophic muscle.