FINALIST 2026
Eva has a rare liver disease called progressive familial intrahepatic cholestasis type 3 (PFIC3), which over time causes severe damage. A liver transplant is the only curative treatment, but this is a major operation and has a risk of complications – and there is a shortage of donor livers. Action Medical Research is funding work to develop gene therapy for PFIC3. If successful, this could ultimately lead to a safer and better treatment option for children with this life-threatening condition.
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