NewsMETABOLISM: CLINICAL AND EXPERIMENTALMonday, March 9, 2026 · March 9, 2026
Efficacy and safety of sepiapterin versus sapropterin in patients with phenylketonuria: Results from the Phase 3, randomized, crossover, open-label, active-controlled AMPLIPHY trial.
WHY IT MATTERS
Recent peer-reviewed research on Phenylketonuria that may be relevant for patients and caregivers.
AMPLIPHY is the first Phase 3 study comparing sepiapterin versus sapropterin in children and adults with phenylketonuria (PKU). AMPLIPHY was an international, Phase 3, two-part, open-label study in participants with PKU aged ≥2 years. Participants responsive to sepiapterin (60 mg/kg...
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