Duchenne muscular dystrophy is a congenital disease affecting young boys almost exclusively. The condition leads to severe muscular dysfunction and death. In the past, this condition has been treated with corticosteroids, which are not very effective, and often introduce their own set of side effects which can be more problematic.
Researchers at Washington University School of Medicine in St Louis are experimenting with the use of rapamycin-laden nanoparticles injected into patients to help alleviate the symptoms of Duchenne’s. So far, researchers have introduced nanoparticles with rapamycin (an immunosuppressant drug) into the blood stream of mice with the condition. The results have been promising, with many of the symptoms of Duchenne’s being alleviated in the mouse models as well as improved cardiac function in the mice as the result of improved blood flow.