• FDA Awards Capricor’s Duchenne Therapy CAP-1002 Rare Pediatric Disease Designation
  • The Challenging Job of Representing the Rare Disease Community in Washington
  • PPMD Designates Gillette Children’s Specialty Healthcare a Certified Duchenne Care Center
  • Sarepta Launches Exondys 51 Managed Access Program in Europe, North and South America
  • Translarna Beneficial for Patients in Transitional Stage of Nonsense Mutation Duchenne MD, Study Finds
  • MDA Awards Neurologist Johanna Hamel $130,000 Clinical Research Training Fellowship
  • Everest to End Duchenne to Hold Second Fundraising Trek to Mount Everest Base Camp
  • FDA Grants Orphan Designation to Mallinckrodt’s MNK-1411 for DMD
  • Problems in RNA Processing Seen to Lead to Congenital Myotonic Dystrophy in Early Study
  • Most Common Congenital Muscular Dystrophy in UK Is MDC1A, 12-year Analysis Shows
  • A Guide to Medical IDs and How to Pick the Right One for You
  • Hand Braces During Sleep Can Help Preserve Muscle Function of Boys with Duchenne MD, Study Suggests