• Researchers Develop Stem Cell Line to Study Sanfilippo Syndrome Type B
  • Gene Therapy ABO-101 Granted FDA’s Fast Track Status for Sanfilippo Syndrome Type B
  • Alpha-Synuclein Not Responsible for Early Symptoms of Sanfilippo Type A, Study Shows
  • Exome Data That Include Unknown Genetic Variants May Better Estimate Sanfilippo Type B Incidence, Study Says
  • Team Sanfilippo Planning to Launch Phase 2 Trial Testing Trehalose as Sanfilippo Treatment
  • Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
  • Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
  • Siblings of Terminally Ill Children Need More Support, Young Website Founder Says
  • NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
  • Phase 2/3 Trial for MPS IIIA Gene Therapy Candidate LYS-SAF302 Starts Dosing Patients
  • WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
  • Hernias, Joint Rigidity, Skeletal Alterations Among First Signs of MPS, Study Finds