Tag Archives: Exon skipping

  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. BioMarin Quarterly Update to the Duchenne Community

    Posted on November 20, 201512:47 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 »

  3. 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 »

  4. 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 »

  5. 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 »

  6. 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 »

  7. Mack Brown, Jack Ingram, Matthew McConaughey, Vince Young…and The Revell Family, Champions Fighting to Cure Duchenne

    Posted on May 13, 20154:49 pm

      For some people it takes a trophy or medal to denote a champion. For those in the fight against Duchenne Muscular Dystrophy, there are all kinds of champions and you won’t know them by their trophy. Duchenne parents Tim and Laura Revell are such champions but they didn’t plan it this way. When their Read more »

  8. 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 »

  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 »

  10. Sarepta Therapeutics Reports Long-Term Outcomes through 168 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy

    Posted on January 12, 20155:03 pm

    Sarepta Therapeutics released the 168 week data for their exon skipping drug, eteplirsen, this morning.  During a three year period, 7-13 year old boys with Duchenne (the age range of the boys who began the 201 study) would be expected to slow down considerably and many expected to lose ambulation entirely.  After more than three Read more »

  11. Cure Duchenne Has Funded All Types of Duchenne Research

    Posted on September 23, 20145:15 pm

    CureDuchenne is proud to have supported all types of Duchenne research over the past 10 years. That is why CureDuchenne has been a leader in funding exon skipping, stop codon, rare and duplications mutations, utrophin upregulation as well as anti-fibrotic and anti-inflammatory therapies during the past decade. Click here for full image. CureDuchenne’s focus is Read more »

  12. Drisapersen, Etiplersen, GSK, Prosensa and Sarepta, a Positive Week for Duchenne

    Posted on April 11, 201311:09 pm

    Thanks to GSK, Prosensa and Sarepta and, the landscape is changing for the patients and families that are suffering from Duchenne, a severe muscle disease that shortens the life expectancy and quality of life for approximately 300,000 boys and young men worldwide. Even though these potential treatments that are currently in the news have not Read more »

  13. FDA Webinar on Accelerated Approval Summary

    Posted on February 21, 20131:28 am

        CureDuchenne was honored to host a webinar today, in collaboration with PPMD and MDA to discuss accelerated approval for drugs for rare diseases.   Dr. Robert Temple, director of the Office of Medical Policy of FDA’s Center for Drug Evaluation and Research presented detailed information on the different options available to speed the approval of Read more »

  14. Cure Duchenne Supports Duchenne Duplication Mutation Research with Dr. Kevin Flanigan

    Posted on December 28, 20126:42 pm

      Exon duplication mutations have been an area of great interest to CureDuchenne, and last year, CureDuchenne funded research specifically directed toward these mutations in Duchenne.  Dr. Kevin Flanigan at Nationwide Children’s Hospital has successfully completed the first phase of his research (please see below). CureDuchenne will continue to fund his work in 2013. The three Read more »

  15. 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 »