• Patient Advocacy Groups Worldwide Plan Events to Mark Rare Disease Day, Feb. 28
  • Twice-a-day Cerdelga Is Slightly Better Than Once-a-day Regimen for Gaucher, Trial Shows
  • Possible Treatment for Neuropathic Gaucher Disease, Arimoclomol, Shows Potential in Early Study
  • Rare Disease Groups, Patients Differ on ‘Right to Try’ Bill Before US Congress
  • MRE Analysis of Liver Stiffness Helps Monitor Disease Severity in GD Type 1 Patients
  • Impaired Eye Movements Correlate with Clinical Status of GD Type 3, Study Finds
  • Some GD Type 1 Patients May Benefit from ERT-SRT Therapy Combo, Case Report Shows
  • NORD Fundraiser Sets 7,000-Mile ‘Bike, Run or Walk’ Goal for 7,000 Rare Diseases
  • Ambroxol May Have Multiple Effects on Neurons in Gaucher, Parkinson’s Diseases
  • Brazilian Researchers Establish Nursing Diagnosis Guidelines for Gaucher Disease
  • Researchers Identify New Therapeutic Target for Gaucher-Linked Bone Disease
  • Survey May Help Understand Clinical Features of Gaucher Disease Type 3