Statement from MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D.: “This is exciting news for our community. MDA is committed to bringing safe and effective treatments and cures to kids and adults living with neuromuscular diseases as quickly as possible. There is an urgent and unmet need for effective DMD treatments, and we applaud the FDA for recognizing the need to expedite the review process. We expect great things from Santhera and are thrilled to see positive data from their Phase III trial with Raxone/Catena.”
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.