Standing up from a chair, climbing stairs, brushing one's hair – all can be a struggle for people with a rare form of muscular dystrophy that causes progressive weakness in the shoulders and hips. Over time, many such people lose the ability to walk or to lift their arms above their heads. This form of the disease, called limb girdle muscular dystrophy (LGMD), affects a few thousand people nationwide in the United States. As other rare illnesses, LGMD tends not to attract much attention from researchers and funding agencies, so progress toward developing therapies has been slow. But a team at Washington University School of Medicine in St. Louis that identified a subtype (LGMD1D) of the disease in 2012 has shown that lithium improves muscle size and strength in mice with this form of muscular dystrophy. The new findings, published in the April 2019 issue of Neurology Genetics, could lead to a drug for the disabling condition. The open-access article is titled “Lithium Chloride Corrects Weakness and Myopathology in a Preclinical Model of LGMD1D.” "There are no medications available for people with limb girdle muscular dystrophy, so we are very excited to have a good therapeutic target and a potential therapy," said senior author C. Chris Weihl, MD, PhD, a Professor of Neurology who treats people with muscular dystrophy at the university's Neuromuscular Disease Center. "This has been an amazing project. It all began when we diagnosed a patient with muscular dystrophy of unknown cause. Genetic sequencing then helped us identify a new subtype, and we've been able to take that all the way through to a possible therapy."
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