Small molecules may open the door to new therapies for Duchenne muscular dystrophy

The researchers identified a group of small molecules that could open the door to the development of new therapies for Duchenne muscular dystrophy (DMD), an untreated disease that causes devastating muscle weakening and loss. Molecules tested by the University of Pennsylvania’s Perelman School of Medicine team facilitated the repression of a specific gene, utrophin, in […]