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A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)

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A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)

Status: Not yet recruiting
Phase of Trial: Phase IV

Latest Information Update: 04 Nov 2024

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At a glance

  • Drugs Avalglucosidase alfa (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions
  • Sponsors Genzyme-Corporation-Medtronic-(JV)

    • Most Recent Events

    • 04 Nov 2024 New trial record

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