Circulating levels of ceruloplasmin are reduced in ~95% of patients presenting with WD. This is measured in the serum or plasma by immunologic methods or by testing for oxidase activity against ...

A minority of patients taking Ultragenyx Pharmaceutical’s Wilson disease gene therapy UX701 have come off standard-of-care ...

Ultragenyx Pharmaceutical RARE reported positive updates from the Stage 1 cohorts of its phase I/II/III Cyprus2+ study ...

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today reported that the Phase 1/2/3 Cyprus2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in ...

The aim of medical treatment for WD is to remove the toxic deposit of copper from the body to produce a negative copper balance, and to prevent its reaccumulation. Successful therapy is measured ...

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today reported that the Phase 1/2/3 Cyprus2+ study of its UX701 gene therapy has demonstrated meaningful clinical ...