This symposium, presented at SIMD 2024, discusses the results of the Phase 3 APHENITY trial, which investigated the safety and efficacy of sepiapterin in patients with phenylketonuria (PKU)
Society for Inherited Metabolic Disorders (SIMD)
The Society for Inherited Metabolic Disorders (SIMD) meets annually to generate meaningful discussions between clinicians and investigators on inborn errors of metabolism to facilitate collaboration. It fosters clinical research in the biochemical and molecular basis of normal and metabolic disorders to raise awareness of these rare conditions.
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This poster, presented at SIMD 2024, discusses the preliminary results from the ongoing APHENITY extension study, which evaluates the safety of sepiapterin and its effect on dietary phenylalanine (Phe) consumption in participants with phenylketonuria (PKU)
This poster, presented at SIMD 2024, evaluates the efficacy and safety data of sepiapterin vs placebo in patients with phenylketonuria (PKU) who were receiving sapropterin dihydrochloride at the time of APHENITY study entry
▼ Sepiapterin is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.
Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.
MED-ALL-PTC923-2500012 | September 2025
MED-ALL-PKU-2500224 | October 2025