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rhPPCA (UX004)

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Company Ultragenyx Pharmaceutical Inc.
DescriptionRecombinant human protein protective cathepsin-A (rhPPCA)
Molecular Target
Mechanism of ActionEnzyme replacement therapy
Therapeutic ModalityBiologic: Enzyme
Latest Stage of DevelopmentPreclinical
Standard IndicationGlycosphingolipid storage disorders
Indication DetailsTreat galactosialidosis
Regulatory Designation

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