Tag Archives: BioMarin

  1. 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 »

  2. 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 »

  3. 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 »

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      I imagine that by now you have heard the news that BioMarin has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) in Europe.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

  5. 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 »

  6. 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 »

  8. 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 »

  9. 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 »

  10. Speak Up for the Duchenne Community!

    Posted on October 14, 201511:18 pm

      After waiting for a very long time, the Duchenne community now has clarity on the Advisory Committee meetings dates for BioMarin and Sarepta.  BioMarin’s date is November 24, 2015 and Sarepta’s date is January 22, 2016.  Please remember, these dates are tentative until they actually appear on the Federal Registry.  Both of the Ad Read more »

  11. 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 »

  12. Do You kNOW Your Duchenne?

    Posted on September 2, 20154:35 pm

      When our son, Hawken was diagnosed with Duchenne almost 13 years ago, my husband and I felt there couldn’t be too many things worse for a family to experience.  In the years since, I’ve met hundreds of Duchenne families and I’ve realized that many parents had a worse experience than we did. Hawken was Read more »

  13. Duchenne Community: Let Your Voice Be Heard!

    Posted on August 27, 20156:27 pm

      This week the FDA accepted Sarepta Therapeutics’ New Drug Application for eteplirsen with priority review. This follows on the June FDA’s acceptance of BioMarin’s NDA for drisapersen, also with priority review. The next step for both of these drugs is an FDA Advisory Committee meeting in November where both companies will demonstrate why these Read more »

  14. CureDuchenne’s Early Funding Projects Take a Big Step Forward Today

    Posted on June 29, 20153:58 pm

      FDA Accepts NDA for Drisapersen, the First-Ever Treatment for Duchenne Muscular Dystrophy to Reach this Milestone in the U.S., and Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen  It is a big day for the Duchenne community.  It is also a proud moment for CureDuchenne.  Today, BioMarin announced that the U.S. Food and Read more »

  15. 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 »

  16. Treatments on the Horizon

    Posted on May 21, 20153:41 pm

      The Duchenne community is celebrating another promising step toward an approved treatment. On May 19 Sarepta Therapeutics announced plans to submit a rolling NDA for eteplirsen with the final NDA submission landing in the FDA’s hands by mid-year.   CureDuchenne is proud to be part of the support and funding for eteplirsen. We collaborated Read more »

  17. Passion for Physical Therapy and Duchenne Care

    Posted on May 8, 20154:14 pm

    Guest blog by Jennifer Wallace, physical therapist and project manager of CureDuchenne Cares. Yesterday was an important day for me since it was my last day working in a local pediatric physical therapy clinic.  Although I have loved my time at this clinic, I have a strong passion for working with my Duchenne patients.  I Read more »

  18. Hope and Gratitude: Moving Closer to a Cure for Duchenne

    Posted on April 28, 20157:13 pm

    As I type this, my heart is filled with hope. A horizon that we could barely see 12 years ago has arrived. When our son Hawken was diagnosed with Duchenne Muscular Dystrophy 12 years ago, we felt lost and scared. By forming CureDuchenne we took on the challenge of finding a cure for this horrible Read more »

  19. 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 »