Publication: Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY extension study
This research article reports the interim results from the phase 3 APHENITY extension study, which evaluates long-term treatment with sepiapterin in phenylketonuria (PKU) patients
- Learn more about the long-term treatment of PKU with sepiapterin
- Understand the effectiveness of long-term treatment with sepiapterin in these interim results from the ongoing, open-label, phase 3 APHENITY extension study
- Gain an overview of the safety profile of sepiapterin in this study
MED-US-CORP-2500008 | November 2025
Van Spronsen F, Peters H, Margvelashvili L et al. Genet Med. 2026;12;28(4):101683
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MED-US-CORP-2600001 | February 2026
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