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

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Casazza K, Cologna SM, Berry-Kravis E, Jarnes J, Porter FD.

J Inherit Metab Dis. 2025 Sep;48(5):e70075. doi: 10.1002/jimd.70075.

2025

PMID

40814945

https://doi.org/10.1002/jimd.70075

PSP

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