Abstract
Wilson's disease (WD), also called hepatolenticular degeneration, is an autosomal recessive copper dysfunction disorder that is among the few treatable neurogenetic disorders. The main goals for controlling WD are to lower copper intake and encourage copper removal. Medical treatments and low-copper diets are available, but many problems remain. For example, current treatments cannot fix copper metabolism and are unable to cross the blood–brain barrier. Furthermore, severe side effects have been experienced by many WD patients. Because lifelong therapy is required for this disease, adherence to medical therapy and best practices for monitoring and personalizing therapy continue to evolve; some of these issues are being addressed in ongoing studies. Additionally, novel chelating agents, gene therapies using adeno-associated viruses, and variant-specific therapies that may improve neurological outcomes are in development. Here, we review the latest treatment innovations that may play an essential role for WD patients in the future. • Wilson's disease (WD) is one of the few neurogenetic diseases that can be treated. • Current treatments neither can fix the copper metabolism nor cross the blood-brain barrier (BBB). • Besides, severe side effects have been experienced by many WD patients. • Novel chelating agents, gene therapy, and variant-specific treatments are in development.
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