• 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
  • Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
  • Video Games Connect Chronically Ill Children Isolated at Home, Hospital
  • Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
  • RANK Pathway Components May Be Markers of Bone Disease in Gaucher, Study Says
  • Rare Diseases Clinical Research Network Opens Online Survey on COVID-19