Gene therapy for wilson disease: a closer look with PET scans
Knowledge-focused
ENROLLING_BY_INVITATION
This study uses special PET scans to see how gene therapy (UX704) changes copper levels in the liver and body of people with Wilson disease. Only 5 patients who already received the gene therapy will join. The goal is to understand if the therapy helps the liver handle copper bet…
Sponsor: Thomas Damgaard Sandahl • Aim: Knowledge-focused
Last updated May 11, 2026 20:53 UTC