Serum neurofilament light protein as a biomarker in Niemann-Pick disease, type C1.

Genet Med Open · 2025 · PMC12351337 · PMID 40821948

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highly significantno p-value reported

The results show moderate but highly significant correlations between log(serum NfL) levels and measures of both clinical severity (ANO and ASIS) and disease burden (5-and 17-domain NPC NSS), all with FDR-adjusted P ≤ .0001.

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