This is awesome news. DMD is a horrible, horrible disease and it looks like we are that much closer to a cure for it.A new experimental drug appears to improve the muscle function of boys with muscular dystrophy, increasing the distance the boys can walk in six minutes compared to when they began taking it a year before.
Those results are “incredibly promising,” says Lee Sweeney, chairman of physiology at the University of Pennsylvania’s Perelman School of Medicine, who studies muscular dystrophy and was not involved in the study. “They don’t ever improve once they start going down. The fact you can improve them is not something that happens normally. That’s not random.”
The most common form of the disease, Duchenne muscular dystrophy, affects only 13,000 American boys; girls almost never get it because is caused by a defect of a gene on the X chromosome, of which girls have a second copy. The new medicine, eteplirsen, is targeted at the 13% of those boys whose muscular dystrophy is caused by a certain type of mutation. Without treatment, patients’ muscles continually weaken, leaving them wheelchair-bound; they frequently die by their 30s.
Eteplirsen, if successful, will be a huge victory for muscular dystrophy patients and researchers, who have never had a treatment that might make any of these kids get any better.