Today, the first safe and effective treatment for Duchenne muscular dystrophy—Eteplirsen—was approved.
This is a proud day for every member of our Duchenne Community who worked for this moment and this historic victory—the experts who weighed in with the FDA, the community who rallied, and most of all the 12 boys in this trial who persevered through five long years. THANK YOU. We wouldn’t be here without your contributions.
The Race to Yes isn’t done. We’ll keep in touch.
—The Race to Yes Team