Possibly Helpful Therapeutic for Myotonic Muscular Dystrophy; Drug Is Targeted to Inhibit TWEAK Pathway

A doctor who was one of the discoverers of the gene responsible for myotonic muscular dystrophy has now identified a therapeutic that could modify progression of muscle damage and muscle dysfunction associated with the disease - issues that cause patients significant disability and deterioration in quality of life. The potential treatment is an experimental drug currently being evaluated for treating other conditions, such as rheumatoid arthritis. In lab tests, mice with myotonic dystrophy that were given the treatment had better muscle function in tests such as running on a treadmill and had improved grip strength. In addition, their muscles became healthier and, notably, many even lived longer. While more testing needs to be done, Mani S. Mahadevan, M.D., of the University of Virginia (UVA) School of Medicine, is hopeful about the drug's potential in humans as well. "The nice thing about this therapy is that we know that it's already been shown to be safe, because clinical trials have already been done with it for other conditions. That's a big, big hurdle that's been overcome," Dr. Mahadevan said. "With a lot of drugs, the problem is that once you do these proof-of-concept studies, the drugs need to be developed a lot further, refined, and tested for safety and efficacy. But a lot of that work has been done, so therefore we can leapfrog the development of this therapy so that it can be moved into clinical trials sooner." Dr. Mahadevan, of UVA's Department of Pathology, has been conducting pioneering research into the causes of myotonic dystrophy, the most common form of muscular dystrophy, for more than two decades. His work revealed that the condition is caused by an expanding piece of DNA - a mutation that grows worse with each generation.
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