• Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe
  • Looking at Holiday Self-isolation in a Positive Light
  • DM1 Patients Asked to Join Natural History Study Aiming for Better Clinical Trials
  • Expressing Our Negative Emotions to Find Positive Ones
  • Phase 1 Trial of Oral EDG-5506 for Becker MD Enrolling at Texas Site
  • Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry
  • Q&A: Meet the Muscular Dystrophy Association’s New CEO and President
  • MDA Launches Tool to Make Clinical Data More Accessible to Researchers
  • Saying Goodbye to Eye Strain, Thanks to Blue-light Glasses
  • AMO-02 for Congenital DM1, Entering Pivotal Trial, Gets Rare Pediatric Disease Status
  • How I’m Getting Out of My 2020 Funk
  • Ultragenyx and Solid Biosciences Partner on DMD Gene Therapy