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. |
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| 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 |
| ISSN: | 1573-2665 |
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| DOI: | 10.1002/jimd.70075 |
