A note from Debra

Eteplirsen, Drisapersen, the FDA and Dedicated Duchenne Moms

Posted on March 26, 2014 5:46 pm

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 Duchenne boys 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.

Drisapersen Update with Prosensa

Posted on March 19, 20146:13 pm

Prosensa will present a status update on Drisapersen and its follow-on compounds in a patient focused webinar hosted by United Parent Projects Muscular Dystrophy (UPPMD) on Tuesday, March 25 at 1 p.m. ET. If you have any questions regarding recent Prosensa/drisapersen updates or any other topic related to the company’s Duchenne portfolio that you would Read more »

Prosensa Announces 48-Week Data from a U.S. Phase II Placebo-Controlled Study of Drisapersen in 51 DMD Boys

Posted on March 17, 201410:25 pm

        Clinically meaningful improvement from 24-week treatment period was maintained for 24 weeks after drisapersen administration ceased Chicago, IL, March 17, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today reported encouraging 48-week  data from its Read more »