News

A study entitled “Left ventricular systolic function and the pattern of late-gadolinium-enhancement independently and additively predicts adverse cardiac events in muscular dystrophy patients” published in the September issue of the Journal of Cardiovascular Magnetic Resonance report two new independent predictors for adverse cardiac events in Duchenne and…

A new study presented at the EuroSpine Annual Meeting on October 1 – 3 in Lyon, France reported the successful placement of a single rod with hybrid fixation to correct scoliosis in children with Duchenne’s muscular dystrophy. Duchenne muscular dystrophy (DMD) is a genetic disease caused by mutations in…

Beverly Hills, California-based Capricor Therapeutics, Inc. has just announced plans to explore the potential of using one of their leading pipeline products indicated for heart disease, for the treatment of complications from an extremely rare muscle-wasting disease called, Duchenne Muscular Dystrophy (DMD). This specialized biotechnology company has…

Muscular dystrophy patient Robert Ryan is a launching a Youtube channel dedicated to his fight against the disease, called “The Vidiot,” the name his uncle gave him when they started playing video games together. Both suffer from muscular dystrophy, and since Ryan spends most of his time in a…

While the CDC does not have enough data on current numbers of patients with Duchenne/Becker muscular dystrophy (DBMD), it is estimated that one out of every 5,600-7,700 males aged 5-24 years old in the US are affected by this rare muscle-wasting disease. Noticeable symptoms occur…

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…

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…