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Prospective, Open-label, Single-arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory Support

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Trial Profile

Prospective, Open-label, Single-arm, Exploratory Study of the Effect and Safety of rhGAA in Patients With Advanced Late-onset Pompe Disease Who Are Receiving Respiratory Support

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 09 Nov 2021

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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

  • 15 Jul 2009 New trial record

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