Can a new drug protect the developing brain in kids with PKU?
Disease control
Recruiting now
This study aims to see if long-term treatment with a drug called sepiapterin can help preserve normal brain development and thinking skills in children with phenylketonuria (PKU). It will enroll 56 children, from toddlers up to age 16, who have PKU. Researchers will track changes…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 23, 2026 15:17 UTC