A European group of researchers successfully used a combination of genetic correction and stem cells to treat Duchenne muscular dystrophy (DMD) in mice with the disease. The compound they used for the genetic correction was developed by Judith van Deutekom, then at Leiden (Netherlands) University Medical Center, with MDA support.
DMD results from the loss of the muscle protein dystrophin because...
In this issue: An MDA-supported gene therapy safety trial gets federal approval to move forward ** Sudden cardiac death found in people with MMD2, and those with MMD1 often require a pacemaker ** Etanercept shows promise in mysasthenia gravis
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