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. |
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| 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 |
| ISSN: | 1573-2665 |
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| DOI: | 10.1002/jimd.70194 |
