Wilson's disease at a glance

Wilson'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