Tiny trial uses PET scans to peek at gene therapy for wilson disease
Knowledge-focused
ENROLLING_BY_INVITATION
This study will use special PET scans to see how gene therapy (UX704) changes copper distribution in the bodies of 5 people with Wilson disease. The goal is to understand if the therapy helps the liver handle copper better. It is an early, observational imaging study, not a test …
Sponsor: Thomas Damgaard Sandahl • Aim: Knowledge-focused
Last updated Jun 26, 2026 15:22 UTC