• Family Known as Face of Sanfilippo Syndrome Keeps Working for a Cure
  • Phoenix Nest Gains Rights to Potential Gene Therapy for Sanfilippo Syndrome Type C
  • Study Gathers Parents’ Experiences of Caring for Children with Mucopolysaccharidosis
  • Shire’s Investigational ERT Shows Limited Benefits for Sanfilippo Type A Children in Phase 2b Trial
  • Combined Behavioral, Pharmacological Therapy Seems to be Best in Treating MPS Disorders
  • #NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
  • Sanfilippo Type A Causes Toxic Buildup of Several Molecules in Various Brain Regions, Mouse Study Shows
  • Experts Release Recommendations for Future Sanfilippo Syndrome Trials
  • Sarepta and Lysogene Sign Licensing Deal for LYS-SAF302, Potential Gene Therapy for Sanfilippo Type A
  • Lower Temperatures Improve NAGLU Activity in MPSIIIB Patients’ Cells, Study Shows
  • Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments
  • Later Stages of Sanfilippo Syndrome Type A Associated with Severe Decline in Communication, Daily Living, Socialization and Motor Skills, Study Reveals