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Disease Burden and Pharmacological Treatment Patterns in Children and Adults With Phenylketonuria: A Real-World Matched Cohort Study.

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Title: Disease Burden and Pharmacological Treatment Patterns in Children and Adults With Phenylketonuria: A Real-World Matched Cohort Study.
Authors: Longo N; Department of Human Genetics, Division of Clinical Genetics, University of California, Los Angeles, California, USA., Burton BK; Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA., Vaghela S; Moderna, Inc., Cambridge, Massachusetts, USA.; HealthEcon Consulting, Inc., Ancaster, Ontario, Canada., Mu F; Analysis Group, Boston, Massachusetts, USA., Wang A; Analysis Group, Boston, Massachusetts, USA., Smith J; Analysis Group, Boston, Massachusetts, USA., Reichert E; Analysis Group, Boston, Massachusetts, USA., Simpson RB; Analysis Group, Boston, Massachusetts, USA., Sikirica V; Moderna, Inc., Princeton, New Jersey, USA., Perera S; Moderna, Inc., Cambridge, Massachusetts, USA.
Source: Journal of inherited metabolic disease [J Inherit Metab Dis] 2026 May; Vol. 49 (3), pp. e70194.
Publication Type: Journal Article
Journal Info: Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22Longo+N%22">Longo N</searchLink>; Department of Human Genetics, Division of Clinical Genetics, University of California, Los Angeles, California, USA.<br /><searchLink fieldCode="AU" term="%22Burton+BK%22">Burton BK</searchLink>; Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Vaghela+S%22">Vaghela S</searchLink>; Moderna, Inc., Cambridge, Massachusetts, USA.; HealthEcon Consulting, Inc., Ancaster, Ontario, Canada.<br /><searchLink fieldCode="AU" term="%22Mu+F%22">Mu F</searchLink>; Analysis Group, Boston, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Wang+A%22">Wang A</searchLink>; Analysis Group, Boston, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Smith+J%22">Smith J</searchLink>; Analysis Group, Boston, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Reichert+E%22">Reichert E</searchLink>; Analysis Group, Boston, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Simpson+RB%22">Simpson RB</searchLink>; Analysis Group, Boston, Massachusetts, USA.<br /><searchLink fieldCode="AU" term="%22Sikirica+V%22">Sikirica V</searchLink>; Moderna, Inc., Princeton, New Jersey, USA.<br /><searchLink fieldCode="AU" term="%22Perera+S%22">Perera S</searchLink>; Moderna, Inc., Cambridge, Massachusetts, USA.
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  Data: <searchLink fieldCode="JN" term="%227910918%22">Journal of inherited metabolic disease</searchLink> [J Inherit Metab Dis] 2026 May; Vol. 49 (3), pp. e70194.
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        Value: 10.1002/jimd.70194
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      – TitleFull: Disease Burden and Pharmacological Treatment Patterns in Children and Adults With Phenylketonuria: A Real-World Matched Cohort Study.
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              M: 05
              Text: 2026 May
              Type: published
              Y: 2026
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