Tag Archives: Sarepta Therapeutics

  1. 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 »

  2. 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 »

  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. The Wait for An Approved Drug for Duchenne Continues

    Posted on May 25, 20161:57 pm

      The wait continues.  Sarepta Therapeutics announced this morning that the FDA will not complete the review of the eteplirsen new drug application by the PDUFA date.  Considering the thousands of lives that could be affected by a decision on eteplirsen, we understand and appreciate the care the FDA is taking in its review of Read more »

  5. Sarepta Advisory Committee Meeting

    Posted on April 22, 201611:45 pm

      As I prepare to travel to Monday’s Advisory Committee meeting I continue to be disappointed by the FDA Peripheral and Central Nervous System Drugs Advisory Committee’s initial review of Sarepta’s Duchenne muscular dystrophy drug eteplirsen. Despite this news we need to remember The FDA’s initial reaction is just that – an initial response – Read more »

  6. 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 »

  7. 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 »

  8. 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 »

  9. 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 »

  10. 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 »

  11. 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 »

  12. 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 »

  13. 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 »