Statement from MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D.: “We are thrilled to see this continued progress from the FDA. OPMD is a rare and debilitating muscular dystrophy for which there is currently no cure or approved therapy, so this is particularly encouraging news. We continue to support the FDA and applaud them for recognizing the need to expedite the review process of Cabaletta, and other potentially lifesaving treatments. MDA remains committed to bringing safe and effective treatments and cures to kids and adults living with muscular dystrophy and related 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.