1. 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 »

  2. NORD Rare Impact Awards

    Posted on May 18, 20165:13 pm

      NORD hosted the 2016 Rare Impact Awards to bring together the rare disease community and to honor and celebrate outstanding achievements.  Last night, Debra Miller, founder of CureDuchenne, was one of the esteemed honorees who was recognized for moving us forward in the fight against rare diseases.  Congratulations Debra for your tireless effort to Read more »

  3. Happy Mother’s Day

    Posted on May 8, 20164:55 pm

      From one mom to another, I wish you a Happy Mother’s Day from the bottom of my heart. As I think about the most wonderful gift in my life, my son Hawken, I am filled with an all-consuming joy. His humor, laughter and innate kindness surprises and encourages me every day. And that’s what Read more »