An experimental drug can effectively cure Duchenne muscular dystrophy in mice by correcting a genetic defect and allowing healthy muscles to develop, researchers reported this week in a paper published online by the journal Nature.
Preliminary studies in humans with muscular dystrophy and cystic fibrosis show that the drug is safe and well-tolerated and hint that it might work for them as well, according to the team headed by H. Lee Sweeney, chairman of the Department of Physiology at the University of Pennsylvania School of Medicine.
The drug has the potential to cure a host of genetic diseases caused by the same mutation, Sweeney said.
"There are literally thousands of genetic diseases that could benefit from this approach," he said. "What's unique about this drug is it doesn't just target one mutation that causes disease, but a whole class of mutations."
Wonderful news. A class of drugs that could treat both MD and cystic fibrosis.