Tag Archives: clinical trial

  1. We need your help to advance a treatment to clinical trials for rare, Duplication 2 mutations!

    Posted on December 15, 20166:56 pm

      CureDuchenne has always been focused on finding and funding treatments to help EVERYONE affected with Duchenne, and to TREAT THE WHOLE DISEASE.  We are very proud of our comprehensive portfolio of research projects and we continue to be the research leader for the Duchenne community. Over the past five years we have pioneered duplication Read more »

  2. Pfizer Clinical Trial Update

    Posted on December 8, 20164:33 pm

      CureDuchenne is pleased to share the following updates from Pfizer pertaining to the on-going multicenter Phase II clinical trial of the investigational compound, domagrozumab (PF-06252616) in boys with Duchenne muscular dystrophy (DMD). Domagrozumab is an experimental, infused, anti-myostatin monoclonal antibody.  Myostatin is a naturally occurring protein in muscles that helps control muscle growth; it Read more »

  3. 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 »

  4. 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 »

  5. 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      I imagine that by now you have heard the news that BioMarin has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) in Europe.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 »

  6. Update on Phase 3 Tadalafil Trial

    Posted on February 3, 20167:08 pm

    We are sharing the following information from Lilly on their Phase 3 Tadalafil Trial   We are writing to share disappointing news about our Phase 3 study of tadalafil in approximately 330 patients with Duchenne muscular dystrophy (DMD). We recently completed analysis of the initial data from the placebo controlled, double-blind period of the trial, Read more »

  7. 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 »

  8. Welcome Dr. Jak Knowles

    Posted on September 16, 20153:46 pm

    We couldn’t be prouder that CureDuchenne has played a critical role in the development of what we hope will be the first-ever pharmaceutical treatments for Duchenne muscular dystrophy. Investments by CureDuchenne in Prosensa (now BioMarin) and Sarepta Therapeutics have helped advance two drugs through clinical trials that await FDA approval in the fall of 2015. Read more »

  9. BioMarin Update on Duchenne Muscular Dystrophy Program

    Posted on April 13, 20159:35 pm

    CureDuchenne is pleased to share the following communication update from BioMarin to the Duchenne community regarding developments of their exon skipping program for exon 51. CureDuchenne has provided extensive support in developing this program throughout the years.  We are delighted that boys will begin redosing.   We would like to update the Duchenne community on Read more »