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Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment.

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Title: Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment.
Authors: Casazza K; Purity Health, Columbia, Tennessee, USA., Cologna SM; Department of Chemistry, University of Illinois Chicago, Chicago, Illinois, USA., Berry-Kravis E; Departments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Rush University Medical Center, Chicago, Illinois, USA., Jarnes J; University of Minnesota, Department of Pediatrics, Minneapolis, Minnesota, USA., Porter FD; Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, Bethesda, Maryland, USA.
Source: Journal of inherited metabolic disease [J Inherit Metab Dis] 2025 Sep; Vol. 48 (5), pp. e70075.
Publication Type: Journal Article; Review
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="%22Casazza+K%22">Casazza K</searchLink>; Purity Health, Columbia, Tennessee, USA.<br /><searchLink fieldCode="AU" term="%22Cologna+SM%22">Cologna SM</searchLink>; Department of Chemistry, University of Illinois Chicago, Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Berry-Kravis+E%22">Berry-Kravis E</searchLink>; Departments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Rush University Medical Center, Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Jarnes+J%22">Jarnes J</searchLink>; University of Minnesota, Department of Pediatrics, Minneapolis, Minnesota, USA.<br /><searchLink fieldCode="AU" term="%22Porter+FD%22">Porter FD</searchLink>; Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, Bethesda, Maryland, USA.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Wiley%22">Wiley </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>7910918 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>1573-2665 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2201418955%22">01418955 </searchLink><i>NLM ISO Abbreviation: </i>J Inherit Metab Dis <i>Subsets: </i>MEDLINE
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        Value: 10.1002/jimd.70075
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        Text: English
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      – TitleFull: Biomarker Validation in NPC1: Foundations for Clinical Trials and Regulatory Alignment.
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              M: 09
              Text: 2025 Sep
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