Eteplirsen, Drisapersen, the FDA and Dedicated Duchenne Moms

Congratulations to the Race to Yes organizers (Tracy Seckler, Jenn McNary, Christine McSherry, Mindy Leffler, and many others) and the Duchenne community for their passionate and successful campaign to petition the White House to urge the Food and Drug Administration (FDA) to use  the Accelerated Approval pathway for approval and access to safe, effective therapies for the treatment of Duchenne Muscular Dystrophy.

CureDuchenne has been an early supporter and investor in exon skipping technology, in both Sarepta (AVI) and Prosensa.  Today we continue to work with other organizations, the FDA and sponsor companies to get both eteplirsen and drisapersen and all safe and effective drugs approved for the treatment of Duchenne.

We appreciate the tremendous media attention this campaign has given to Duchenne, and are encouraged by the positive reports from parents whose sons’ participated in exon skipping trials for eteplirsen, (such as Jenn’s son Max) and drisapersen, (such as Ryan Dunne in Colorado).

Many those impacted with Duchenne who participated in both the eteplirsen and drisapersen clinical trials reported  stabilization or improvement during their treatment and we support all efforts to provide access to these drugs for all boys that can benefit.