• Gaucher Type 1 Therapy AVR-RD-02 Granted EU Orphan Drug Status
  • Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos
  • Efforts Continue Beyond Awareness Month to Expand Newborn Screening Panels in US
  • Potential SRT for Neuronopathic Gaucher Shows Efficacy in Early Study
  • Fatty Liver Common, Linked to Scarring, in Type 1 Gaucher, Study Finds
  • Activities Underway for Newborn Screening Awareness Month
  • Same But Different Photo Contest Celebrates People With Rare Diseases
  • Type 3 Gaucher Patients at Greater Risk of Parkinson’s as They Age, Case Series Suggests
  • Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input
  • Centogene, Evotec Expand Collaboration to Develop New Gaucher Treatments
  • EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US
  • New Streaming Channel Showcases Rare Disease Films