• Enzyme Replacement Therapy, BMN-250, Shows Promise as Treatment for Sanfilippo Type B, Study Finds
  • Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
  • Approved Therapy for Bladder Pain Being Repurposed to Potentially Treat MPS
  • ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration
  • Physicians Recommend Early Surgery to Correct Spinal Issues in MPS Patients
  • FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
  • NAGLU Enzyme Replacement Therapy Fails to Improve Clinical Outcomes, Trial Results Show
  • Abeona Will Include Early-stage MPS IIIA Patients in ABO-102 Gene Therapy Trial
  • Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
  • New Stem Cell Lines Derived From MPSIIIB Patient May Help Improve Preclinical Research
  • 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