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An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen

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Trial Profile

An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen

Status: Completed
Phase of Trial: Phase IV

Latest Information Update: 01 Feb 2016

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At a glance

  • Drugs Alglucosidase alfa (Primary)
  • Indications Glycogen storage disease type II
  • Focus Therapeutic Use
  • Sponsors Genzyme Corporation

Most Recent Events

  • 01 Feb 2016 New trial record

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