A drug that works like a “molecular patch” to bypass faulty genes has brought new hope to children with a devastating muscle-wasting disease.
For the first time, research has shown it is possible to counteract the effects of Duchenne muscular dystrophy (DMD) throughout the body.
Seven out of 19 young patients aged five to 15 responded to the injected drug in an early-stage clinical trial.
DMD is a hereditary disease that affects one in 3,500 newborn boys, with around 100 new cases diagnosed in the UK each year.
It is marked by a lack of dystrophin, a vital muscle protein, leading to the progressive breakdown of muscle cells.
By the age of 12, boys with the disease become unable to walk and by the late teens or early 20s the condition may become life threatening as breathing is compromised.
While not providing a cure, the treatment has the potential to reduce the symptoms of DMD enough to prevent severe disability and allow a reasonable quality of life.
Dr Marita Pohlschmidt, director of the Muscular Dystrophy Campaign, said: “We have fought to find a treatment for this devastating condition for the past 50 years.
“Today we can say with real confidence that we’re going to win that battle. Parents of these boys can have real hope for the future.”
Results of the Medical Research Council-funded trial are published online in The Lancet.