• Approved Bladder Pain Therapy Being Repurposed for Lysosomal Storage Disorders
  • NGF President and CEO Pays Tribute to ‘Scientific Legend’ in Gaucher, Other Metabolic Storage Disorders
  • Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
  • Blood Cells Could Help Screen Ambroxol’s Efficacy in GD Type 3
  • ERT Helps Partially Restore Recycling Process in Cells from Gaucher, Fabry Patients, Study Shows
  • ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration
  • Cerezyme and Cerdelga Equally Effective in Treating Gaucher Type 1, Small Study Reports
  • FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
  • Case Report of Multi-Generational GD Highlights Importance of Cascade Screening
  • Gaucher Disease May Rarely Include GI Symptoms, Case Report Highlights
  • Computer Simulation Identifies Treatments More Likely to Correct GBA Transport in Gaucher Patients, Study Says
  • Key to Early Diagnosis May Lie in Response to Chronic Anemia Treatment, Case Report Suggests