Tag Archives: Clinical trials

  1. Better Designed Clinical Trials Now

    Posted on October 31, 20168:25 pm

      As a parent of a son with Duchenne I have been frustrated that the data presented to the FDA does not match the patient experience.  I personally know so many patients who have been in recent clinical trials that are doing much better than expected and are seeing a slower progression of their disease Read more »

  2. Learn. Engage. Empower.

    Posted on October 10, 20169:15 pm

      Caring for a child with Duchenne can be daunting, complicated, and often times, isolating. But it doesn’t have to be. On October 22, CureDuchenne Cares will hold its inaugural Family Summit bringing together Duchenne families, medical providers and researchers for a daylong event focused on education and fellowship. The goal of this summit is Read more »

  3. We are “All in Building a Bridge over Duchenne”

    Posted on September 21, 20164:02 pm

      Guest blog by Tammy and Scott Henegar Our family’s journey with Duchenne muscular dystrophy started in March 2011 when our 4 ½ year old adventurous and sweet young boy was diagnosed with the heart breaking diagnosis that no parent should have to face.  We were told that Braedan would be facing a life of Read more »

  4. First Drug Approved for Duchenne: A Beacon of Hope for the Duchenne Community

    Posted on September 20, 20165:08 pm

      My husband Paul and I have dreamed of this day since our son Hawken was diagnosed with Duchenne almost 15 years ago! We are overwhelmed with joy to announce that the FDA has officially granted accelerated approval for Eteplirsen for the treatment of Duchenne Muscular Dystrophy. This is the first approved drug-therapy and a Read more »

  5. BioMarin Update for the Duchenne Community: 7th July, 2016

    Posted on July 7, 20163:00 pm

      BioMarin is providing a further update to the Duchenne Community following our announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on compounds BMN 044, BMN 045 and BMN 053. Update for Patients Receiving Drisapersen, BMN 044, 045 or 053 in Clinical Read more »


    Posted on June 9, 20165:45 pm

      Gene therapy proposes a promising approach in treating Duchenne, placing corrected genes into cells that have missing or damaged genes. Although gene therapy is still relatively early, CureDuchenne is encouraged that Bamboo Therapeutics is planning a human clinical trial for 2017.  As always, we need to be mindful of the difficulty of delivering leading Read more »

  7. BioMarin Update for the Duchenne Community: 01 June 2016

    Posted on June 2, 20162:59 pm

      We are sharing the following update from BioMarin. We would like to update the patient community in light of the recent announcement regarding the EMA marketing application. BioMarin is discontinuing clinical and regulatory development of drisapersen as well as the other three follow-on products: BMN 044, BMN 045 and BMN 053 Why have we Read more »

  8. Capricor Therapeutics Announces First Patient Treated In HOPE-Duchenne Clinical Trial

    Posted on February 22, 20161:19 pm

      CureDuchenne is pleased to see our biotech partner Capricor Therapeutics progress with their HOPE-Duchenne clinical trial. Capricor announced today the treatment of the first patient with Duchenne muscular dystrophy-related cardiomyopathy with CAP-1002 in the HOPE-Duchenne Phase I/II clinical trial. The patient was treated at the Cincinnati Children’s Hospital Medical Center under the direction of Read more »

  9. 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 »

  10. 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 »

  11. 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 »

  12. Capricor Therapeutics – Webinar on Upcoming Clinical Trial for Cardiac Indication in Duchenne

    Posted on June 18, 201510:34 pm

      There has been a lot of recent Duchenne research news lately.  As a community, we are glad to see a variety of research programs moving forward with positive results. This provides hope for all those that live with Duchenne and their families. Duchenne is a complex disease and will require a variety of treatments Read more »

  13. CureDuchenne – Clinical Trials – FDA

    Posted on December 7, 201212:45 am

    Following the long awaited news of Sarepta’s phase IIB results for eteplirsen and continued Phase III progress of GSK/Prosensa’s drisapersen, as well as the recent developments with PTC Therapeutics and Summit PLC among others, CureDuchenne took the initiative to reach out for patients and advocacy groups alike to begin a dialogue with the FDA and Read more »