Duchenne Muscular Dystrophy (DMD) is a debilitating genetic disorder that eventually leaves patients in a wheelchair. In a new study, researchers have found that an existing cancer drug shows promise in slowing the progress of DMD by changing the type of muscle fibers to be more resilient.
DMD occurs as the result of a gene mutation on an X chromosome, which reduces the production of a protein called dystrophin. Without it, muscle cells become fragile and easily damaged, leaving patients with progressively weaker muscle function. Eventually, the disease progresses to muscles involved in heart and lung function, resulting in a shortened life expectancy.
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While there’s currently no cure, treatments to slow the progression of the disease are in development, such as drug combinations or boosting a related muscle-strengthening protein. Gene therapy could one day correct the disorder, with experiments on mice, dogs and pigs proving promising.
Read more: New Atlas
