Wilson's disease at a glanceWilson's disease (WD) is a rare genetic disorder of impaired copper metabolism that causes severe hepatic and neurologic symptoms. It is lethal without a treatment.
Unmet medical needs:
- With current care, neurologic manifestations improve in only 55 % of the patients, and a few patients deteriorate further which suggests irreversibility; 50% patients have residual neurological symptoms despite treatment
- Side effects may be serious, especially with chelators that may induce paradoxical neurologic deterioration at treatment onset
- Lifelong constraining oral therapy for Wilson's disease leads to poor adherence to treatment (up to 50% patients), with the risk of decompensation with acute liver failure and death
- Liver transplant and associated immunosuppression carry their own risks