• NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
  • Cerdelga Has Favorable Long-term Safety Profile in Patients with Type 1 Gaucher Disease, Pooled Trial Data Show
  • Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
  • Non-motor Symptoms Preceding Parkinson’s Onset Prevalent in Gaucher Type 1, Study Finds
  • ‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy
  • Gaucher Type 3C Evaluations Should Include Heart, Lungs, Researchers Say
  • Case Report of Sisters with Gaucher Underscores Importance of Early Diagnosis
  • MicroRNA Inactivation Fails to Reduce Inflammation, Increase Survival in Zebrafish Models of Gaucher
  • Saposin C Protein Key in Regulating Gaucher Disease Severity, Mouse Study Shows
  • Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
  • Preclinical Data Has AVR-RD-02 Gene Therapy on Track for First Trial in Humans
  • Gene Activity Profiling Reinforces Inflammation’s Role in Gaucher Disease