• Cerezyme ‘Dramatically’ Eased Severe Liver and Lung Problems in Gaucher Patient, Case Study Reports
  • Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
  • New GBA Mutation May Be Linked to Perinatal Lethal Gaucher Disease, Case Report Suggests
  • Gain Therapeutics and Partners Win €1.4M Grant to Develop Treatments for Gaucher and Other Diseases
  • Ways Needed to Ease High Cost of Gaucher Care to Patients and State in Iran, Study Says
  • Psychologist Living with Gaucher Strives to Be ‘Role Model of Success’
  • Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
  • Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
  • Scoring Swallowing Problems Helps Stage Neurological Decline in Type 2 GD Infants
  • Scientist Gets Funding to Create 3-D Bone Tissue Model for Gaucher
  • Centogene Rolls Out Free Biomarker App for Gaucher Patients
  • Pentosan Polysulfate May Reduce Inflammation in Gaucher, Fabry, Study Suggests