1. An Update for the Duchenne Community

    Posted on June 24, 201612:30 am

      We are sharing the following update from Sarepta Therapeutics.   Sarepta Therapeutics is updating the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study 4045-301), as posted on clinicaltrials.gov. [Identifier: NCT02500381] ESSENCE will serve as a confirmatory study for eteplirsen if it is granted accelerated approval. Post-marketing confirmatory studies Read more »

  2. A Father’s Day Letter to My Fellow Duchenne Dads

    Posted on June 19, 20163:52 pm

    From Paul Miller Another Father’s Day is here and I wish you all a meaningful day filled with memories that will last a lifetime. As I think about the most meaningful gift in my life, my son Hawken, I am overcome with pride. To me, he is perfect. His smile, his laughter and his unwavering Read more »

  3. BioMarin Update for the Duchenne Community: 17 June 2016

    Posted on June 17, 20165:56 pm

      BioMarin is providing a further update to the Duchenne Community in light of recent announcements of withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on products BMN 044, BMN 045 and BMN 053. Is there any Update for Patients Receiving Drug in Clinical Trials? 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 »

  5. 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 »

  6. Going Backwards to Go Forwards

    Posted on June 1, 201612:27 am

      I imagine that by now you have heard the news that BioMarin has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) in Europe. Like you, I am heartbroken and sad to see this happen. From a business standpoint, BioMarin’s protective step makes sense. The company needs to regroup to find a better way forward. Read more »