1. CureDuchenne Urges Regulators to Approve Eteplirsen, Stresses the Importance of Multiple Drugs in the Pipeline

    Posted on January 18, 20168:17 pm

      The Duchenne community continues its fight to get an approved treatment for Duchenne. The FDA released Briefing Document on eteplirsen, the drug for Duchenne muscular dystrophy submitted for approval by Sarepta Therapeutics Inc.  Although the review is harsh, we urge the Advisory Committee and the FDA to listen to the patient voice when making Read more »

  2. BioMarin DMD Update for Patient Groups

    Posted on January 15, 20167:02 pm

      We wanted to share the following information from BioMarin in regards to yesterday’s news on the FDA decision on Kyndrisa (drisapersen).     Dear Patient Group Representative, As you are likely aware, yesterday, we announced that the FDA issued a Complete Response Letter to BioMarin for our new drug application for Kyndrisa (drisapersen) for Read more »

  3. CureDuchenne-Funded Trial for Cardiac Therapy

    Posted on January 13, 201611:54 pm

      Capricor Therapeutics is actively recruiting participants for a clinical trial for a potential cardiac cell therapy for those with Duchenne muscular dystrophy.  CureDuchenne funded Capricor to support this trial that addresses heart disease, which is the primary cause of death for those with Duchenne.     The Halt cardiOmyopathy ProgrEssion in Duchenne (HOPE-Duchenne) is a Read more »