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Company Amicus Therapeutics Inc.
DescriptionRecombinant human acid alpha-glucosidase (rhGAA)
Molecular Target Acid alpha glucosidase (GAA)
Mechanism of ActionEnzyme replacement therapy
Therapeutic ModalityBiologic: Protein
Latest Stage of DevelopmentPhase I/II
Standard IndicationPompe's disease
Indication DetailsTreat Pompe's disease
Regulatory Designation

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