• Fat ‘Floaters’ Impairing Vision in GD3 Patient Highlight Need for Monitoring, New Therapies, Case Study Reports
  • FDA Grants Orphan Drug, Rare Pediatric Disease Designations to PR001 Investigational Gene Therapy
  • FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
  • Thermo Fisher Develops Test to Screen Genes Linked to Gaucher, Other Hereditary Disorders
  • With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
  • IGA, Kantar Partner to Build Global Gaucher Registry for Type 2 and 3 Patients
  • Gaucher Mouse Model Mimics Broad Set of Symptoms to Aid in Therapy Discovery, Study Says
  • Case Report Identifies New GBA Gene Mutation in Woman with Gaucher Type 1
  • Targeted Gene Therapy Prevents Neurological Symptoms, Extends Survival in Gaucher Mouse Model, Study Shows
  • Careful Monitoring for Lung Involvement Urged for Gaucher Children, Even Absent Symptoms, Study Reports
  • ‘Floaters’ That Impair Vision May Help Track GD3 Severity, Small Study Suggests
  • Avrobio Cleared to Expand to the U.S. its Gene Therapy Trial for Gaucher Disease