• Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
  • Gene Therapy ABO-102 Preserves Cognitive Development in Young Children with Sanfilippo Type A, Interim Data Show
  • Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
  • Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
  • Lysogene Creates Scientific Advisory Board to Advance Gene Therapy in Sanfilippo, Other Disorders
  • Heart Problems Less Common, Milder in Sanfilippo Than Other MPS Forms, Taiwanese Study Finds
  • Study Emphasizes Needs of Caregivers of Children with Sanfilippo Type B
  • European Initiative Targets Diagnosis, Treatment of Rare Diseases
  • Scientists Develop Method to Gather CSF Samples in Mice to Study Sanfilippo Biomarker
  • World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
  • Heparan Sulfate Can Collect in Some Patients’ Kidney Cells, Mouse Study Finds