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Muscular Dystrophy Symptom Reversible?

Nov. 15, 2007 -- Scientists today reported that they have reversed symptoms of myotonic dystrophy -- adults' most common type of muscular dystrophy -- in lab tests on mice.

"This work should provide hope and encouragement to people with myotonic dystrophy and their families," University of Rochester neurologist Charles Thornton, MD, says in a news release.

Muscular dystrophies include more than 30 genetic diseases that weaken and break down skeletal muscles that control movement.

Thornton's team created a compound called a morpholino that serves as a patch to make up for a genetic glitch linked to myotonic dystrophy.

The scientists injected the morpholino into the leg muscle of mice with myotonic dystrophy. A single shot of the morpholino reversed symptoms for up to eight weeks, the study shows.

The morpholino hasn't been tested in people.

But the tests in mice prove the notion that "a fundamental aspect of this genetic disease can be reversed even after it is very well established," says Thornton.

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

The findings appear in today's online edition of The Journal of Clinical Investigation.