Real-world experience with sepiapterin in phenylketonuria: A single-center retrospective analysis.

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Title: Real-world experience with sepiapterin in phenylketonuria: A single-center retrospective analysis.
Authors: Vucko ER; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA. Electronic address: evucko@luriechildrens.org., Arduini K; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA., Becker K; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA., Kozek A; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA., Kurkjian B; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA., McGrath G; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA., Meza A; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA., Schirmacher S; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA., Shim S; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA., Smith M; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA., Prada CE; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA., Burton BK; Edwards Family Division of Genetics & Rare Diseases, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA; Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.
Source: Molecular genetics and metabolism [Mol Genet Metab] 2026 Aug; Vol. 148 (4), pp. 110153. Date of Electronic Publication: 2026 May 29.
Publication Type: Journal Article
Journal Info: Publisher: Academic Press Country of Publication: United States NLM ID: 9805456 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1096-7206 (Electronic) Linking ISSN: 10967192 NLM ISO Abbreviation: Mol Genet Metab Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1096-7206
DOI:10.1016/j.ymgme.2026.110153