Duchenne muscular dystrophy (DMD) affects approximately 1 out of every 3,500 male infants (about 20,000 new cases a year). This form of muscular dystrophy results from mutations in the dystrophin gene that lead to an absence of dystrophin in muscle cells, allowing these cells to be easily damaged. Boys living with DMD experience devastating muscle weakness that affects the skeletal muscles, heart, and respiratory muscles. Symptoms of DMD typically develop prior to age 5, and by age 12 most patients are confined to a wheelchair. Currently, there is no cure for DMD and young men with this disease rarely live beyond their early 30s. Progress is being made in understanding DMD; however, there are still many challenges that exist to improve the treatment for this disease.
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