Today, very little is known about Limb-Girdle Muscular Dystrophy (LGMD), a hereditary disorder that affects the muscles proximal to the body and causes progressive dystrophy. It is estimated to affect roughly one in 14,500 to one in 123,000 individuals — numbers that could very well be undercounts as LGMD has…
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Catabasis to Present DMD Candidate Treatment at International Congress of the World Muscle Society
CAT-1004, a candidate treatment for Duchenne muscular dystrophy (DMD) being developed by Catabasis Pharmaceuticals, will be featured in a poster presentation at the International Congress of the World Muscle Society, taking place in Berlin, Germany between October 7 and 11. Catabasis Pharmaceuticals is a drug developer currently focused on the development of clinical…
Research on a newly discovered type of RNA molecule could help eventual treatment of degenerative muscle and brain diseases such as the most common form of muscular dystrophy that starts in adulthood. Human genetic information — the “blueprint” of our constitution and that of and almost all other living…
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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