DENVER - A local family is celebrating the news that a prescription drug company will push forward for a larger study for the Muscular Dystrophy drug eteplirsen.
Ryan Dunne is nine. He has Duchenne Muscular Dystrophy.
In March, more than 100,000 Coloradans and people across the country signed a petition on Change.org urging the Food and Drug Administration to use the Accelerated Approval pathway for approval and access to safe, effective therapies for Duchenne Muscular Dystrophy.
Monday, Massachusetts-based Sarepta Therapeutics said it plans to file a new drug application with the FDA and will push forward with a larger “confirmatory” study that will allow more boys access to eteplirsen and give regulators more robust data about its safety and effectiveness, according to the Washington Post.