• Avrobio Pauses Dosing in Phase 1/2 Trial of Gene Therapy for Gaucher
  • Common Antibody Abnormalities in Gaucher Not Linked to Disease Severity, Study Finds
  • NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum
  • Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19
  • Cerdelga Leads to Sustained Benefits in Type 1 Gaucher Patients, Study Says
  • Younger Children With GD1 More Susceptible to Poorer Health-related Quality of Life, Study Suggests
  • Rare GD1 Genetic Profile Associated With Mild Symptoms, Study Finds
  • Specific Lipid Traced to Multiple Myeloma Risk in Type 1 Gaucher Patients
  • Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds
  • Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare
  • Israeli Gaucher Clinic Treats Arabs With Disease Prevalent Among Ashkenazi Jews
  • Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29