• FDA Grants Rare Pediatric Disease Status to Myonexus’ MYO-101 for LGMD 2E
  • Myoda (Sarconeos) Earns FDA’s Orphan Drug Designation for Duchenne MD
  • ‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35
  • #ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say
  • Sarepta, Myonexus Collaborating to Advance Gene Therapies for LGMD
  • 12 Things to Pack for Your Hospital Stay
  • New Exon 51 Skipping Therapy in Phase 1 Safety Trial Explained by Sarepta CEO in Webinar
  • Approved Anti-inflammatory Seen to Increase Muscle Strength and Function in DMD Mice in Study
  • PPMD Marks 10th Year of Duchenne Registry, Largest Repository of DMD Patient Data
  • Capricor Recruiting Duchenne Patients for Phase 2 Trial of CAP-1002
  • Sarepta Therapeutics Launches ‘Route 79, the Duchenne Scholarship Program’
  • Acceleron’s ACE-083 Therapy Candidate for FSHD Earns FDA’s Fast Track Designation