A blood-based cell therapy slows muscle deterioration in children and young people with Duchenne muscular dystrophy, according to a phase III trial
Duchenne muscular dystrophy (DMD) is a serious genetic condition that causes progressive muscle weakness and atrophy, including of the heart. There is currently no cure, and it mainly affects boys and young men. A cell therapy called Deramiocel could slow down muscle weakness in children and young people with advanced-stage DMD, according to a phase III clinical trial published in The Lancet, the first of its kind. The study involved 106 children and young people aged between 10 and 22 with advanced DMD. According to the results, after one year, participants who received the drug lost mobility in their arms 54 per cent more slowly than those in the placebo group, although this did not result in a clear difference in the heart’s ability to pump blood.