• Blood Test Plus Algorithm Might Help to More Easily Spot Gaucher Type 1
  • Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients
  • AVR-RD-02 Gene Therapy Still Showing Efficacy in 1st Treated Patient in Trial
  • Pandemic Won’t Stop Rare Disease Day on Feb. 28
  • FDA Grants Orphan Drug Status to M6P Gene Therapy
  • NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns
  • Newly Found RNA Molecules May Be Biomarkers for Disease Progression
  • COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say
  • Cerezyme Effective Over 20 Years in Easing GD1 Symptoms, Data Show
  • NORD’s Caregiver Respite Program Continues Through Pandemic
  • Cerdelga Seen To Safely, Effectively Treat GD1 After Long-term ERT Use
  • NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum