Duchenne muscular dystrophy research at the University of Florida got a major boost with the award of $7.5 million in National Institutes of Health funding to study the use of magnetic resonance imaging in determining the natural progression of the disease.
UF scientists will assess whether MRI technology can be used as a precise, noninvasive measure of muscle tissue in children with Duchenne muscular dystrophy. Understanding how the disease affects muscle tissue could help facilitate the testing of new therapies in clinical trials, researchers say.
Duchenne muscular dystrophy affects about one of every 3,500 to 5,000 boys born each year in the United States, according to the Centers for Disease Control and Prevention. The disease causes the muscles that control movement to progressively weaken and lose the ability to regenerate after an injury, eventually replacing critical muscle tissue with fat and collagen. By age 12, many patients need a wheelchair. As the disease advances, the heart and respiratory system are affected and patients often die of cardiorespiratory failure in their 20s.
"The lack of a reliable assessment tool for measuring muscle function in patients with Duchenne inhibits the transfer of new therapies from the lab to clinical trials," said the study's lead investigator KristaVandenborne, Ph.D., an associate dean for research and planning at the UF College of Public Health and Health Professions and chair of the department of physical therapy. "MRI allows you to look at the structure of a muscle tissue in a very objective way with a large amount of detail. Our goal is to develop MRI as a tool to see the progression of the disease, but more importantly, to determine if a new treatment is effective or not, giving researchers rapid feedback about potential new drugs."
The study is funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the National Institute of Neurological Diso
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University of Florida