• Avalglucosidase Alfa Improves Breathing, Mobility in Late-onset Patients in Phase 3 Trial
  • Coalition Will Address Racial Disparities in Rare Disease Communities
  • Reproductive Options if You Are a Pompe Disease Carrier
  • COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
  • Genetic Testing for Pompe Disease: What to Expect
  • Lumizyme-Myozyme Given Early and at Higher Dose May Be Best for Infantile-onset Pompe
  • What Is Genetic Counseling and Why Is It Important in Pompe Disease?
  • Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
  • Video Games Connect Chronically Ill Children Isolated at Home, Hospital
  • Plant-based ERT May Hold Promise in Pompe Disease, Small Study Suggests
  • Unity and EU-wide Efforts Focus of Online Rare Disease Meeting