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A Global Prospective Observational Registry of Patients With Pompe Disease

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A Global Prospective Observational Registry of Patients With Pompe Disease

Status: Recruiting
Phase of Trial: Phase IV

Latest Information Update: 28 Feb 2025

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At a glance

  • Drugs Alglucosidase alfa (Primary) ; Avalglucosidase alfa (Primary) ; Cipaglucosidase alfa (Primary) ; Miglustat (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions
  • Sponsors Amicus Therapeutics

    • Most Recent Events

    • 01 Mar 2024 Planned End Date changed from 20 Nov 2033 to 20 Dec 2034.
    • 01 Mar 2024 Planned primary completion date changed from 20 Nov 2033 to 20 Dec 2034.
    • 01 Mar 2024 Status changed from not yet recruiting to recruiting.

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