Wilson disease is a genetic disorder that prevents the removal of extra copper in the body. Without treatment, the disease can cause life-threatening organ damage due to the copper built up in the ...

Since the observation that injections of 2,3-dimer-captopropanol (BAL) benefit the neurologic symptoms 8 and, in 1 case, 9 may have benefited a patient in the "preneurologic" stage, it is of the ...

DNA helix. VTX-801 is an investigational, novel rAAV-based gene therapy that delivers a shortened version of the ATP7B transgene. The Food and Drug Administration (FDA) has granted Fast Track ...

The US Food and Drug Administration (FDA) has approved oral trientine tetrahydrochloride (Cuvrior, Orphalan) for the treatment of adults with stable Wilson's disease who are decoppered and tolerant to ...

Wilson’s disease is a rare genetic disorder in which copper accumulates in the body. It usually affects the liver and the brain, but can also involve the kidneys, the heart and the eyes. Symptoms are ...

Researchers report that they have conducted promising preclinical experiments on a compound that could be used to treat Wilson's disease and possibly other disorders -- including certain types of ...

For decades, Wilson’s Disease has evaded comprehensive treatment. The wide-ranging physical and neurological symptoms of the condition have upended afflicted patients’ lives, with no potential cure in ...

A Toronto developer of treatments based on artificial intelligence said today it has successfully used its own AI drug discovery platform to identify its first therapeutic candidate, a therapy for ...

The Hah1 metallochaperone protein is implicated in copper delivery to the Menkes and Wilson disease proteins. Hah1 and the N-termini of its target proteins belong to a family of metal binding domains ...