A cell therapy called deramiocel could slow muscle weakening in boys and young men with advanced Duchenne muscular dystrophy (DMD) and may also slow heart damage in those who already have heart muscle disease, a Phase III clinical trial published in The Lancet has found.

It is the first Phase III trial of a cell therapy made from donor cells and administered through the bloodstream to treat a genetic disease, and the first such trial in boys and young men whose DMD is already advanced. The therapy is grown from heart cells that were donated for transplant but could not be used.

There is no cure for DMD, a serious genetic condition that causes the muscles, including the heart, to gradually weaken and waste away. It almost exclusively affects boys and young men because the gene involved sits on the X chromosome. As the disease progresses, most patients lose the ability to walk and come to depend on their arms and hands for everyday tasks and independence.