Potential new treatment for Duchenne muscular dystrophy

Posted on Wednesday, July 27th, 2011 in Health

new treatment for Duchenne muscular dystrophyResearchers have developed a potential treatment for some cases of Duchenne muscular dystrophy.

Duchenne muscular dystrophy is a muscle wasting condition which effects one in 3,500 newborn boys. Approximately 100 new cases arise in the UK every year.
The condition causes the patient’s muscle to waste away throughout their life and can leave many in a wheelchair by the age of ten. By the age of 30, it begins to be life threatening as it starts to damage the muscles which regulate breathing.

A gene in the X chromosome is responsible for making a protein called dystrophin. The Duchenne muscular dystrophy is where this gene is damaged or mutated and results in a loss of dystrophin production.

The new treatment works by injecting a patient with tailored pieces of antisense RNA which cuts out the damaged or mutated part of the genetic code and allows the creation of a shorter protein. The shorter dystrophin protein is still able to function. In a 12 week trial involving 19 patients, seven children saw a degree of dystrophin production return.

The lead researcher, Prof Francesco Muntoni, indicated that the best result showed a 20% return of dystrophin production. He added: “I’ve worked with patients with Duchenne muscular dystrophy for many years and this is the first time we can say with confidence that we’ve made a significant breakthrough towards finding a targeted treatment.”

Prof Muntoni added that this treatment was tailored for the most common type of the condition which affects 13% of sufferers. He believes a similar treatment can be used to treat the second most common cause which affects 11% of the sufferers.

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