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A prospective, observational real-world safety and effectiveness of Cipaglucosidase alfa and Miglustat in adults with late-onset Pompe disease

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Trial Profile

A prospective, observational real-world safety and effectiveness of Cipaglucosidase alfa and Miglustat in adults with late-onset Pompe disease

Status: Recruiting
Phase of Trial: Phase IV

Latest Information Update: 09 Oct 2024

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At a glance

  • Drugs Cipaglucosidase alfa (Primary) ; Miglustat (Primary)
  • Indications Glycogen storage disease type II
  • Focus Therapeutic Use

    • Most Recent Events

    • 09 Oct 2024 New trial record
    • 04 Oct 2024 According to an Amicus Therapeutics media release, data from this study will be presented at the 29th Annual Congress of the World Muscle Society being held October 8-12, 2024 in Prague, Czechia.

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