Adapted from Schilsky ML, Tavil AS. Wilson disease. In: Schiff's Diseases of the Liver. Philadelphia: Lippincott Williams and Wilkins; 2006:1023–1040. Most practitioners prefer start with trientine.

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

On-track to initiate IND-enabling activities for Wilson’s Disease program in 4Q 2024, with IND or CTA filing expected in 1H 2026 --CAMBRIDGE, ...

Further, with a history of jaundice, she was diagnosed with Wilson’s disease, which was the underlying cause of proximal RTA with isolated phosphaturia and consequent fractures. Standard treatment for ...

Unless you have Wilson's disease, or you know someone who does, you've probably never heard of this rare genetic condition. With non-specific symptoms that mainly affect the brain and the liver, this ...

Last year, AstraZeneca reported that a new drug to treat Wilson disease developed by its Alexion rare diseases unit had hit the mark in a phase 3 trial, setting it on course for regulatory filings.

A minority of patients taking Ultragenyx Pharmaceutical’s Wilson disease gene therapy UX701 have come off standard-of-care drugs, leading the biotech to enroll a new cohort on a tweaked regimen ...

Wilson disease is a rare genetic condition that causes too much copper to collect in the body. Children born with Wilson disease cannot get rid of extra copper from their bodies. The copper buildup ...

Wilson disease is a genetic disorder where the body is unable to properly eliminate excess copper, leading to its buildup in organs like the liver, brain and eyes. This can cause liver damage ...