1. BioMarin Update to the Duchenne Community-November 25, 2015

    Posted on November 26, 201512:52 am

      BioMarin expresses its heartfelt appreciation to the Duchenne patient organizations, families, boys and young men living with Duchenne muscular dystrophy (Duchenne) who voiced their perspectives at the November 24th Food and Drug Administration (FDA) Advisory Committee meeting to consider our New Drug Application (NDA) for drisapersen for the treatment of Duchenne amenable to exon Read more »


    Posted on November 25, 201512:05 am

    CureDuchenne, the California-based nonprofit organization dedicated to finding cures for Duchenne muscular dystrophy, released the following statement from its co-founder and CEO, Debra Miller, following the meeting of the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA). The FDA will consider the committee’s discussions, and has set a Read more »

  3. FDA Advisory Committee Meeting

    Posted on November 20, 20154:50 pm

      We are looking forward to the U.S. Food & Drug Administration (FDA) Advisory Committee meeting on  Tuesday, November 24. This will be an important day for the Duchenne community. The committee members selected by the FDA are set to review clinical data for Drisapersen and hear testimony from patients, doctors and caregivers, the individuals Read more »

  4. BioMarin Quarterly Update to the Duchenne Community

    Posted on 12:47 am

      CureDuchenne is pleased to share the BioMarin Quarterly Update to the Duchenne Community.   It covers important information about next week’s FDA Advisory Committee meeting and other key updates from the company.     November 2015 BioMarin: Quarterly Update to the Duchenne Community Welcome to the second of the BioMarin quarterly updates. As you can Read more »

  5. You are Pioneers Once Again

    Posted on November 17, 20159:22 pm

      We are pleased to share the following letter from the CEO of PTC Therapeutics to the Duchenne community.  CureDuchenne funded PTC Therapeutics back in 2003 and we are proud to have helped advance the development of Translarna.  We look forward to PTC completing their rolling New Drug Application (NDA) with the Food & Drug Read more »

  6. CureDuchenne’s Debra Miller and Jak Knowles Spoke on Venture Philanthropy and Duchenne at Recent Conferences

    Posted on November 16, 20159:44 pm

    CureDuchenne’s success in venture philanthropy has attracted the attention of the medical research community around the world. Last week, CureDuchenne CEO Debra Miller and Vice President of CureDuchenne Medical and Scientific Affairs Jak Knowles spoke at two different conferences about venture philanthropy and how it positively influences otherwise unapproachable biomedical technologies. CureDuchenne has been leading Read more »

  7. Cardiac Trial for Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy

    Posted on November 5, 20154:51 pm

      Did you know that heart failure is very common and often fatal in patients with Duchenne muscular dystrophy? The Halt cardiOmyopathy ProgrEssion in Duchenne (HOPE-Duchenne) is a clinical trial that will evaluate whether an investigational cardiac cell therapy is safe and potentially effective in minimizing scar and improving heart function in DMD associated heart Read more »

  8. Transfer Techniques to Protect Duchenne Patients and Caregivers

    Posted on November 3, 20156:13 pm

      Moving someone who has Duchenne from one location to another is no easy task, especially when considering patient and caregiver safety. Physical therapist,  owner at Duchenne Therapy Network and CureDuchenne Cares program director, Jennifer Wallace approaches this topic in her recent blog video highlighting the importance of using proper techniques when performing transfers. Transfers Read more »