Carmen Bertoni

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...

An MDA grant will help develop RTC13, a drug designed to cause dystrophin protein production in DMD-affected muscle fibers

posted on February 9, 2011 - 11:39am
A new MDA translational research grant for $476,465 over three years will allow Carmen Bertoni at the University of California, Los Angeles (UCLA) to develop RTC13, an experimental compound designed to treat Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) caused by a specific type of flaw in the gene for the muscle protein dystrophin.