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The experimental drug RTC13 caused dystrophin production and functional benefit in Duchenne MD research mice, outperforming similar drugs

posted on June 22, 2012 - 6:00am
An experimental drug called RTC13, designed to treat Duchenne muscular dystrophy (DMD) by restoring production of the muscle protein dystrophin, has shown promise in experiments in dystrophin-deficient mice that have a DMD-like disease. RTC13's MDA-supported developers say they're optimistic about the compound but that refinement of its chemistry and further testing will be needed before it can...

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