Can a daily pill save kids' brains from PKU damage?
Disease control
Recruiting now
This study tests a drug called sepiapterin in 56 children with phenylketonuria (PKU), a genetic condition that can harm the brain if not managed. The goal is to see if starting treatment early can preserve thinking skills like IQ and quality of life over the long term. Children a…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 17, 2026 07:32 UTC