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Reversal of muscle disease

16th November 2007

Scientists at the University of Rochester have been able to reverse one of muscular dystrophy's most important symptoms in laboratory experiments.

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The study, published on the internet by the Journal of Clinical Investigation, found a compound that stopped myotonia (muscle tensing) in mice who had been bred to develop muscular dystrophy.

The most prevalent form of muscular dystrophy in adult sufferers is known as myotonic dystrophy. People with this form of the disease experience progressive muscle wasting and weakness.

Myotonic dystrophy destroys muscles' ability to relax and can affect the hand muscles most severely, resulting in clenched, "locked" hands.

The Rochester scientists came up with a molecule which, when given to mice with the disease, was able to restore a "critical control mechanism" and allowed muscles to relax.

Dr Thurman Wheeler, the team's leader, said that the molecule was very stable once it had been injected into the muscles and had "surprisingly" long-lasting effects.

However, the team said that more work needed to be done in order to make the treatment suitable for testing in human subjects. The scientists' next challenge will be to find an improved method of delivering the molecule through the body.

Researcher Dr Charles Thornton, director of the university's Neuromuscular Disease Center, said: "This work should provide hope and encouragement to people with myotonic dystrophy and their families."

"As we move forward, we should not be content to keep this condition from getting worse. We should set our set our sights on making it better."

 




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