• Abeona Partners With Taysha to Advance ABO-202 as Potential Gene Therapy
  • Same But Different Photo Contest Celebrates People With Rare Diseases
  • Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
  • FDA Names Investigational Therapy BBDF-101 Orphan Drug, Rare Pediatric Disease
  • Good News for CLN1 Disease and a Sprint to the Finish
  • EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
  • Neurogene’s CLN7 Gene Therapy Granted FDA Orphan Drug Designation
  • When Is It OK to Quit Fighting Rare Disease?
  • New Streaming Channel Showcases Rare Disease Films
  • Case Report Describes Rare Case of Newborn Diagnosed With CLN2 Disease
  • URMC Hopes New IDDRC Research Designation Leads to Scientific Advances
  • Your Rare Disease Reading List for Quarantine: Part 2