Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a rare, genetic disorder that affects the muscles of the body causing progressive, muscle wasting and eventual loss of mobility. It primarily affects boys, and is caused by mutations in the gene for the protein dystrophin. Without adequate dystrophin, muscles become weak and eventually waste away. DMD is one of the most common and severe inherited neuromuscular disorders, affecting approximately 1 in 5000 male births. DMD can cause severe disability, reducing the ability of those affected to walk, run, and move normally. It can also affect the heart, breathing, and other muscles in the body. As the disorder progresses, the need for assistance with everyday activities increases and without treatment, most individuals with DMD will require a wheelchair by the time they enter their teens. Currently, there is no cure for DMD, however, recent advancements in gene therapy, coupled with other therapeutic approaches, have greatly increased the life expectancy of those affected by this disorder. Furthermore, there are treatments available to reduce the symptoms and slow the progression of the disease. These treatments include medications, physical therapy, and assistive devices. The research into Duchenne Muscular Dystrophy is ongoing, and the medical community is committed to finding a cure for this disorder. Advances in gene therapy and continued research will remain critical for a successful outcome for those affected by DMD.