• CHOC Children’s Given $8M to Develop Next-Generation Therapies for Pompe Disease
  • ERT vs. SRT – Which Could Be More Effective for Pompe Disease?
  • Amicus Completes Patient Enrollment for Phase 3 Trial of AT-GAA; Remains on Track to File BLA in 2021
  • The Assistance Fund Now Includes Pompe Disease
  • Problems with Diaphragm Evident in Lung Function Tests of LOPD Patients, Study Reports
  • Nerve Damage-induced Muscle Atrophy Coexists with Pompe Disease, Study Asserts
  • MDA Seeks Patient Input for First Pompe Disease Drug Development Meeting in Washington
  • Gene Therapy Takes Center Stage at 2019 NORD Summit
  • Enzyme Replacement Therapy for Pompe Disease – What to Keep in Mind
  • Managing Fatigue in Pompe Disease
  • HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
  • Experts Barry Byrne, Jerry Mendell Lead NORD Webinar on Gene Therapy