• My Daughter Has a Terminal Illness. Now What?
  • The Devastating Phone Call that Confirmed My Sister’s Diagnosis
  • My Daughter’s Journey to a Diagnosis
  • Finding Peace with My Sister’s Diagnosis of Sanfilippo Syndrome
  • Sanfilippo Type A Changes Metabolism of Sugar and Fat Molecules in Brain Regions, Mouse Study Shows
  • NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
  • Sanfilippo Should Be Considered in Landau Kleffner Syndrome Diagnoses, Case Report Suggests
  • Lysogene Announces Goals for Its MPSIIIA Clinical Program
  • Alpha-Synuclein Buildup Doesn’t Weigh on Early Stages of Sanfilippo Type A, Mouse Study Suggests
  • ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
  • Seelos Therapeutics to Begin Phase 2b/3 Trial of Potential Sanfilippo Therapy Trehalose
  • At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing