Tag Archives: NDA

  1. 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 »

  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. 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 »

  4. 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 »

  5. 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 »