MTE #29 Novel Diagnostics and Therapeutics for Wilson Disease

Description

Wilson copper storage disease (WD) research is rapidly evolving. New diagnostic and therapeutic approaches will alter the way the disease is managed in the years ahead. These scientific advances will ensure that patients are receiving accurate diagnoses and effective therapies, thus preventing end-organ injury. Experts review the current guidelines for the diagnosis and treatment of WD and contrast this with emerging novel diagnostics and therapeutics, including gene therapy.

 

Objectives

  • Discuss clinical scenarios highlighting Wilson disease diagnostic challenges.
  • Review Wilson disease therapeutic monitoring, treatment, and alternatives in the setting of nonresponse.
  • Describe novel biomarkers for Wilson disease diagnosis and monitoring.
  • Discuss future therapies for Wilson disease, including gene therapy trials.