• ‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy
  • Scientists Use New Cellular Models to Test BMN 250 Investigational Enzyme Replacement Therapy
  • Amicus Therapeutics and UPenn Expand Gene Therapy Partnership to Lysosomal Disorders
  • Gangliosides, or Sugar-fat Molecules, May Serve As Biomarkers for Sanfilippo, Study Suggests
  • Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
  • New Growth Charts from Birth to Adulthood Can Help Predict Children’s Development, German Researchers Say
  • Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
  • First Sanfilippo Type B Patient Treated with High-Dose ABO-101 Gene Therapy in 2nd Part of Phase 1/2 Trial
  • Tissue Remodeling, Toxic Molecule Accumulation Responsible for Upper Airway Involvement in MPS, Study Finds
  • New Mutation Linked to Sanfilippo Type A Found in Young Chinese Girl
  • 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