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.
Potential benefits of AAV gene therapy, as a novel approach to manage Wilson's disease
- Alternative to current treatments
- Restoration a normal physiological and homeostasis of copper metabolism
- Generation of long-lasting effects by a single IV administration
- Alleviation of the need for repeat oral administration of current treatments and of their associated side effects
- Optimal adherence to treatments, especially in neurologically/ cognitively impaired patients