• 25% of Patients in Spain on ERT Report Missed Doses Due to COVID-19
  • 1st Patient Dosed in Trial of AVR-RD-02, Gene Therapy for Gaucher Type 1
  • National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US
  • Lysogene, Weizmann Institute Team Up on Gene Therapy for Neuronopathic Gaucher
  • Arimoclomol Shows Potential to Aid Gaucher Patients in Phase 2 Trial
  • Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic
  • Long-term Cerezyme Followed by Cerdelga Delayed Bone Loss in Gaucher Type 1 Case Study
  • Two Pro-inflammatory Proteins May Be Biomarkers of Gaucher Type 1
  • Coalition Will Address Racial Disparities in Rare Disease Communities
  • Cerdelga Effective for Gaucher Type 1 in Real-world Setting, Study Shows
  • Liver Health Needs Constant Monitoring in Gaucher Patients, Study Says
  • COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say