1. With Gratitude this Thanksgiving

    Posted on November 23, 20164:53 pm

      Thanksgiving is more than a chance to appreciate what we have. It’s also about taking the time to appreciate the people who help us in our journey, those who share in our hopes and dreams, and our struggles and our pain. For all of you, we are full of gratitude. We are filled with Read more »

  2. Help Support the 21st Century Cures Act and the OPEN ACT

    Posted on November 17, 20164:56 pm

      The final decisions could be made today on the 21st Century Cures Act and the OPEN ACT – a provision that could be transformational for rare disease patients. CureDuchenne signed the letter of support. Despite outreach from constituents, we are still hearing pushback from Sen. Warren on the OPEN ACT. If you could please Read more »

  3. Spotlight on Sandi Preston, PT, CureDuchenne Cares Certified Physical Therapist

    Posted on November 16, 20166:36 pm

      Understanding a patient’s motivation for treatment is the key for a physical therapist to empower that patient for the best outcome. For a patient with Duchenne muscular dystrophy, a terminal disease that deteriorates muscle tissue, this empowerment can be the difference between more cherished ambulatory time or losing their drive for therapy altogether. In Read more »

  4. Marathon Webinar: Update on Deflazacort Access and Regulatory Status

    Posted on November 4, 20164:48 pm

    Thank you to Marathon Pharmaceuticals for providing updated information about deflazacort access and regulatory status during a webinar this week.  The webinar addressed Marathon’s Expanded Access Program for deflazacort and what a potential FDA approval of deflazacort would mean in terms of access to the drug in the U.S.  The webinar has been archived.  https://cc.readytalk.com/play?id=hl12zw. Read more »