NewsGENETICS IN MEDICINE : OFFICIAL JOURNAL OF THE AMERICAN COLLEGE OF MEDICAL GENETICSSaturday, April 11, 2026 · April 11, 2026
Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: Interim results from the phase 3 APHENITY Extension Study.
WHY IT MATTERS
Recent peer-reviewed research on Phenylketonuria that may be relevant for patients and caregivers.
To report interim results from the ongoing, open-label, phase 3 APHENITY Extension Study (NCT05166161), evaluating long-term treatment with sepiapterin in patients with phenylketonuria. Participants received an age-based dose of oral sepiapterin daily; those with mean blood phenylalanine (Phe) level...
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