Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.: “This is very encouraging news for the DMD community, and we hope it means that a treatment is on the horizon. If drisapersen were to receive FDA approval, it would be the first therapy to market in the United States to treat the cause of DMD in a subset of patients. MDA has supported research in exon skipping for DMD since the 1990s and we are more committed than ever to bringing safe and effective treatments and cures to kids and adults living with neuromuscular diseases as quickly as possible.”
The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.