Tag Archives: PTC 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. You are Pioneers Once Again

    Posted on November 17, 20159:22 pm

      We are pleased to share the following letter from the CEO of PTC Therapeutics to the Duchenne community.  CureDuchenne funded PTC Therapeutics back in 2003 and we are proud to have helped advance the development of Translarna.  We look forward to PTC completing their rolling New Drug Application (NDA) with the Food & Drug Read more »

  3. 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 »

  4. 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 »

  5. World Muscle Society Poster Sessions

    Posted on October 10, 20145:32 pm

    WMS Berlin continues to deliver evolving data and novel insights into the treatment, study and diagnosis of Duchenne Muscular Dystrophy.  The location in one of the historical districts for Berlin and German medicine has offered a unique and appropriate setting for keynotes, oral reviews and poster presentations.   Some highlights from the posters over the Read more »

  6. 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 »

  7. 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 »