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Drug hope for muscular dystrophy

5th May 2011

New research has identified a possible treatment for Duchenne muscular dystrophy.

A drug that can boost muscle strength in mice has shown promise as a potential treatment for the condition following research – published in the journal PLoS ONE – and led by Professor Dame Kay Davies, of Oxford University.

Scientists say the discovery may lead to a daily pill to treat all patients with the muscle-wasting disease.

Professor Davies said: “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.”

The inherited condition causes progressive muscle weakness and about 100 boys are born with it in the UK each year. There is no effective treatment.

Oxford University scientists identified the drug by screening therapeutic compounds for the ability to raise levels of a substance that boosts muscle strength.

The substance - utrophin - seems to compensate for the lack of a key protein, dystrophin, which does not work properly in muscular dystrophy.

The most promising candidate, named SMT C1100, was tested in a mouse model of muscular dystrophy. Mice given the drug developed stronger muscles and could run 50% further in exercise tasks.

Professor Max Parmar of the Medical Research Council said: “This study, without necessarily providing us with the final solution, does gives us an important platform from which to move forward and really make a serious progression through clinical trials.”

Preliminary tests conducted in healthy human volunteers raised no safety concerns but scientists acknowledge that further research is required.

 

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