Neurofilament light chain levels correlate with clinical measures in CLN3 disease.

ABSTRACT:

Purpose

CLN3 disease is a neurodegenerative disorder with onset in childhood. It affects multiple functions at different developmental stages. Incomplete understanding of the pathophysiology hampers identification of cell and tissue biochemical compounds reflective of the disease process. As treatment approaches are being explored, more sensitive, objective, quantifiable, and clinically relevant biomarkers are needed.

Methods

We collected prospective biosamples from 21 phenotyped individuals with CLN3. We measured neurofilament light chain (NEFL) levels, a marker of neuronal damage, in cross-sectional CSF and serum samples from individuals with CLN3 and in pediatric non-CLN3 controls using two different assays.

Results

Cerebrospinal fluid (CSF) and serum NEFL levels a

SUBMITTER: Dang Do AN 

PROVIDER: S-EPMC8035240 | biostudies-literature | 2021 Apr

REPOSITORIES: biostudies-literature