Scientists have reversed symptoms of myotonic muscular dystrophy in mice by eliminating a buildup of toxic RNA in muscle cells. The work, carried out by scientists at the University of Rochester Medical Center, Isis Pharmaceuticals Inc. and Genzyme, is published in the August 2 issue of Nature.
Duchenne muscular dystrophy (#DMD) is a muscle-wasting #disease that affects one in every 3500 boys around the world. It is a genetic disease that boys inherit from their mother. Girls can inherit the gene as a carrier and may display mild symptoms, but nothing as debilitating as a boy’s experience. Boys are usually wheelchair bound by their early teens. It was once thought incurable. But today, trials of a #WA-developed drug are under way in America with promising results.