Statement from MDA Scientific Program Officer Amanda Haidet-Phillips, Ph.D.: “We are excited to see that Isis Pharmaceuticals reported positive results from patients who were enrolled in their Phase 2 trial and who continued to receive the drug after the trial ended. SMA is a devastating disease for which there are currently no treatments, and children with type 2 or 3 SMA typically lose function slowly over time. These results show that children with type 2 or 3 SMA treated with ISIS-SMNRx not only did not lose function, but continued to get stronger. Additionally, the drug was safe and well-tolerated even after multiple doses. The results from this trial extension look promising and we are hopeful that there may soon be a treatment available to SMA patients to keep their muscles strong and healthy. We’re very happy to see these promising results as we continue our work to get effective therapies on the market.”
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.