• Gaucher Outcome Survey Patient Registry Provides Global Insights into Disease
  • Abcertin Clinical Data Supports Use as Alternative Replacement Therapy for Type 1 GD
  • Rare Disease Patient Groups Unite to Preserve Orphan Drug Tax Credit in US
  • Baylor Study Links Mutations in Lysosomal Genes to Parkinson’s Disease
  • JScreen Targets Universities to Test Jews for Gaucher Disease, Other Genetic Conditions
  • Baby with Gaucher Type 2 Not Inherited from Mother Detailed in Case Study
  • Children’s National, NORD Partner to Create Rare Disease ‘Centers of Excellence’
  • Why Variations in the Neurological Manifestations of Gaucher Make It Difficult to Diagnose
  • Recently Identified Biomarker Could Be Valid Diagnostic Tool for Gaucher Disease, Mouse Study Suggests
  • #NORDsummit – Alternatives to Large Placebo Trials, Grant Awards Among Ways FDA Supporting Rare Diseases, Chief Says
  • New Method of Diagnosing and Monitoring Gaucher Disease Uses Dried Blood Samples
  • #NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says