UVA Researchers Target Mysteries of Duchenne Muscular Dystrophy

If We Know the Cause, Why Don’t We Have Effective Treatments? Duchenne muscular dystrophy affects 1 in 3,500 boys, stealing their ability to walk by age 12 and typically killing them by their mid-20s. Doctors know what causes the muscle-wasting disease, but there is no cure and, frustratingly, experimental treatments consistently fail. However, a group of University of Virginia School of Medicine & School of Engineering and Applied Sciences researchers is out to change that, with the help of a $2.5 million grant from the National Institutes of Health. The researchers will use the money to build an astonishingly sophisticated computer model of the disease in a bid to unlock the mysteries that have undermined all efforts to develop new treatments. The model will incorporate every aspect of the research process, from the lab mice in which experimental treatments often show promise to the young children in which those treatments subsequently fail. By taking a holistic, “multi-scale” approach, the researchers intend to determine what’s going wrong and pave the way for breakthroughs that are both long-awaited and desperately needed.