• National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
  • UK Renews Puldysa Early Access Program for Duchenne Patients
  • FDA Asked to Approve Casimersen, Exon 45 Skipping DMD Therapy
  • Don’t Feel Bad About Calling Me ‘Inspiring’
  • Coronavirus Relief Fund Created to Help DMD Patients, Families
  • CHMP Recommends Translarna Label Update That Enables Treating DMD Patients Unable to Walk
  • Looking Backward but Living Life Forward With MD
  • Phase 1 Trial of Potential Oral Treatment for LGMD2i Doses 1st Volunteer
  • Dating in the Era of Coronavirus
  • Collaboration Provides Cough Assist Machines to British Columbia Residents With Neuromuscular Disease
  • Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
  • Creativity Allows Me to Surpass Any Physical Limits