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A Multicenter Study to Evaluate and Characterize the Effects of Pharmacological Chaperones in Cell Lines Derived From Blood and Skin Samples From Patients With Pompe Disease

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Trial Profile

A Multicenter Study to Evaluate and Characterize the Effects of Pharmacological Chaperones in Cell Lines Derived From Blood and Skin Samples From Patients With Pompe Disease

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 22 May 2014

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At a glance

  • Drugs Duvoglustat (Primary)
  • Indications Glycogen storage disease type II
  • Focus Pharmacodynamics
  • Sponsors Amicus Therapeutics

Most Recent Events

  • 22 May 2014 New trial record

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