Daily pill for Duchenne muscular dystrophy

A new drug for the muscle-wasting disease Duchenne muscular dystrophy has shown significant benefits in mice, opening the door for clinical trials.

The promising results indicate that a simple, daily pill to treat all patients with Duchenne muscular dystrophy should be possible, whether or not this specific drug formulation makes it all the way through clinical trials without further development.

The study, led by researchers at Oxford University, the University of Bari in Italy and Summit plc with funding from the Muscular Dystrophy Campaign and the Medical Research Council, is published in the journal PLoS ONE.

'We've shown that the drug can dramatically reduce muscle weakness in mice. These results give us everything we need to go forward into initial clinical trials in humans,' says Professor Dame Kay Davies of the Department of Physiology, Anatomy and Genetics at Oxford University, who led the research.

Duchenne muscular dystrophy affects about 100 boys born in the UK each year, or around 1 in every 3,500 male births.

The disease involves a progressive muscle weakness, with the first signs of difficulty in walking seen between the ages of 1 and 3. Boys with the condition are likely to be in a wheelchair by age 12 and live into their twenties or thirties.