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UX004

Ultragenyx Pharmaceutical Inc.

Drug Names(s): rhPPCA, Recombinant human protective protein cathepsin-A

Description: UX004 is a recombinant human protective protein cathepsin-A, or rhPPCA, intended as an enzyme replacement therapy for galactosialidosis, a rare lysosomal storage disease.

Deal Structure: Ultragenyx and St. Jude Children's Research Hosptial
In September 2012, Ultragenyx in-licensed recombinant human protective protein cathepsin-A, or rhPPCA, from St. Jude Childrens Research Hospital.


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