1. Eteplirsen, Drisapersen, the FDA and Dedicated Duchenne Moms

    Posted on March 26, 20145:46 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 Read more »

  2. 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 »

  3. 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 »

  4. Cure Duchenne to Celebrate 10-Year Anniversary at Champions to Cure Duchenne Gala

    Posted on March 12, 20144:23 pm

    CureDuchenne will host our annual Champions to CureDuchenne Newport Beach Gala on Saturday, March 29 at the Balboa Bay Club & Resort. The event will celebrate CureDuchenne’s 10 years of successfully advancing Duchenne research. We’ll honor Doug Freeman, the business leader who helped shape our venture philanthropy model; dine on a delicious meal created by Read more »