Could a daily powder shield Kids' brains from PKU damage?
Disease control
Recruiting now
This study tests whether sepiapterin, a powder taken daily, can preserve intelligence and quality of life in children with phenylketonuria (PKU) when started early in childhood. The trial will follow 56 children for years, measuring IQ and other outcomes. It is an open-label phas…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jul 25, 2026 00:00 UTC