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rhGUS (UX003)

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Company Ultragenyx Pharmaceutical Inc.
DescriptionRecombinant human glucuronidase beta (rhGUS)
Molecular Target
Mechanism of ActionEnzyme replacement therapy
Therapeutic ModalityBiologic: Enzyme
Latest Stage of DevelopmentPhase III
Standard IndicationMucopolysaccharidosis
Indication DetailsTreat mucopolysaccharidosis VII (MPS VII; Sly syndrome)
Regulatory Designation U.S. - Orphan Drug (Treat mucopolysaccharidosis VII (MPS VII; Sly syndrome));
EU - Orphan Drug (Treat mucopolysaccharidosis VII (MPS VII; Sly syndrome))

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