Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.: “Data from this ongoing clinical trial shows that boys with DMD who were treated with HT-100 demonstrated improvements in muscle strength. If these positive trial results are borne out in future studies, this potential therapy may one day be used to improve the function of limb and respiratory muscles in DMD patients, which in turn could improve these boys’ ability to walk, play and move on their own. MDA has contributed more than $1 million toward the development of HT-100 as a therapy for Duchenne muscular dystrophy, supporting early-stage work that informed the laboratory development of the drug and providing funding for the ongoing trial as well. We’re deeply gratified to see these promising results as we continue our work to get effective therapies for DMD on the market and available to the people who need them.”
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.