• ‘You See a Miracle’: Interview with Scientists on Gene Therapy That Saved Boy Dying of EB
  • Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US
  • KB103 Investigational Therapy Receives Clearance from RAC Committee
  • Transplants of Genetically Altered Skin Treats Boy with Epidermolysis Bullosa, Study Shows
  • Potential Gene Therapy for Dystrophic EB, KB103, Named an Orphan Drug by FDA
  • Krystal Biotech Receives $700K Equity-Based Award to Develop Gene Therapy for DEB
  • BERG Receives Partners in Progress Award for Efforts in Treating Epidermolysis Bullosa
  • Children’s National, NORD Partner to Create Rare Disease ‘Centers of Excellence’
  • #NORDsummit – Alternatives to Large Placebo Trials, Grant Awards Among Ways FDA Supporting Rare Diseases, Chief Says
  • #NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
  • New Mutations Account for Genetically Unsolved Cases of EBS, Researchers Say
  • Abeona to Advance Gene Therapy Research at Commercial Manufacturing Facility in Ohio