• Proteins That Regulate Gene with Critical Role in FSHD Development Identified in Study
  • Hockey Player Hosts 8th Annual Golfing Event to Raise Money for CureDuchenne
  • Dating with a Disability: Part 2
  • FDA Names Potential FSHD Therapy GBC0905 an Orphan Drug to Speed Its Development
  • CHMP Opposes Approval of Exondys 51 to Treat DMD in Europe
  • Canada Trails US in Patient Access to Rare Disease Therapies, CORD President Says
  • Duchenne Trials Aren’t Easy on Families or Patients, Even for Those Able to Qualify, PPMD Founder Says
  • Dating with a Disability: Part 1
  • EU’s CHMP Recommends Translarna to Treat Younger DMD Children After FDA Rejection
  • US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases
  • #ECRD2018 – Duchenne Needs ‘Thinking Outside the Box’ to Advance Treatments But Investors Taking Note, Advocate Says
  • #ECRD2018 – Genome Editing Might Be ‘Cure’ for Rare Diseases But Ethical Guidelines Needed, Panel Says