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...
The drug gentamicin increased dystrophin levels in boys with DMD due to a nonsense mutation, but didn't improve strength at the doses used
posted on April 14, 2010 - 3:34am
Results of the MDA-supported trial of gentamicin in Duchenne muscular dystrophy (DMD) were presented Wednesday, April 14, at the annual meeting of the American Academy of Neurology (AAN), held in Toronto.
Levels of the needed dystrophin protein increased in six out of 12 participants who received the drug for six months. No functional improvements were seen.
In this issue: research news on Pompe disease, Becker and Duchenne MDs, Charcot-Marie-Tooth disease, limb-girdle and myotonic MDs, spinal bulbar muscular atrophy, myasthenia gravis and nemaline myopathy