A recent study entitled “Proteasome inhibitors increase missense mutated dysferlin in patients with muscular dystrophy” published in Science Translational Medicine reports increased expression of the protein dysferlin, via inhibiting proteasome activity, may become a new therapeutic treatment for patients with muscular dystrophy. Muscular dystrophies (as…
A group of runners from Los Altos, California are joining efforts to help raise funds to support an eight year-old girl who suffers from Ulrich Congenital Muscular Dystrophy (CMD), a debilitating disease that confines her to a wheelchair and gives her an average life expectancy no longer than young adulthood. Due…
Duchenne muscular dystrophy (DMD), a highly fatal hereditary disorder, affects one in every 3,600 young boys. It commonly begins with muscle weakness and loss in muscle mass. This muscle wasting is eventually replaced by fat and fibrotic tissue, eventually making it difficult to stand and walk, oftentimes by…
The MD-CARE Act, a piece of legislation designed to accelerate research and provide infrastructure needed for the development of new treatments to treat all nine forms of muscular dystrophy, has passed in the Senate by unanimous consent on September 18th. The Muscular Dystrophy Association (MDA), which has been a major advocate for passage of…
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