CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, issued the following statement today regarding the announcement by Prosensa […]
PTC Therapeutics announced this morning that they have received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency, regarding the company’s […]
Dear Patient Group Representative, We trust this letter finds you well. As announced in a press release and during our quarterly update for the financial community […]
Sarepta Therapeutics, Inc. will host a webcast and conference call for the Duchenne muscular dystrophy (DMD) community on Tuesday, May 13, 2014, beginning at 11:00 am EDT (8:00 am […]
Re-dosing plans are underway and 96-week data from an open-label extension study of drisapersen for the treatment of DMD presented at the American Academy of Neurology (AAN) Leiden, The Netherlands, […]
CureDuchenne is pleased to share the following information from Prosensa. Dear Patient Group Representative, As we continue to receive all of the materials, data and know-how from GSK regarding […]
April 28, 2014, 8:32 a.m. EDT CAMBRIDGE, Mass., Apr 28, 2014 (BUSINESS WIRE) — Sarepta Therapeutics, Inc. /quotes/zigman/10874508/delayed/quotes/nls/srpt SRPT +1.17% , a developer of innovative RNA-based therapeutics, will host a webcast and conference […]
Summary from Seeking Alpha Based on updated guidance from the FDA regarding an early approval pathway for eteplirsen, Sarepta Therapeutics (SRPT) plans to file an NDA by the end of 2014. The […]
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 […]