• Ambroxol Plus ERT Can Ease Neurological Symptoms in Some Gaucher Type 3 Patients, Case Study Suggests
  • FDA Asked to Allow Higher Dose of PROO1 Gene Therapy in Gaucher Type 2 Clinical Trial
  • ‘Simple’ Algorithm Seen to Help Diagnose Rare Diseases Like Gaucher Type 3
  • Stem Cell Injections May Treat Gaucher Disease with Brain Involvement, Mouse Study Finds
  • Study Finds Biomarkers That Might Inform Severity of Gaucher Manifestations
  • ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
  • At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
  • Case Report Suggests Misclassification of Pulmonary Hypertension in Gaucher
  • Cerezyme ‘Dramatically’ Eased Severe Liver and Lung Problems in Gaucher Patient, Case Study Reports
  • Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
  • New GBA Mutation May Be Linked to Perinatal Lethal Gaucher Disease, Case Report Suggests
  • Gain Therapeutics and Partners Win €1.4M Grant to Develop Treatments for Gaucher and Other Diseases