Posted on May 23, 20145:59 pm

    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 application for a conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years Read more »

  2. Prosensa Update: Regulatory Discussions Ongoing & Re-dosing on Track

    Posted on May 22, 20144:50 pm

          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 on May 20, we are encouraged by our ongoing interactions with the regulatory authorities, in both the United States and Europe, and remain on track Read more »

  3. Sarepta to Host Webinar Tuesday, May 13

    Posted on May 6, 20144:36 pm

      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 PDT / 5:00 pm CEST). Chris Garabedian, president and chief executive officer, and Edward Kaye, MD, chief medical officer, will provide information and answer questions Read more »

  4. Prosensa Provides Update on Drisapersen

    Posted on May 1, 20141:43 pm

    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, May 1, 2014(GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA) the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, yesterday Read more »