Validation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS.

ABSTRACT:

Objective

To identify preferred neurofilament assays and clinically validate serum neurofilament light (NfL) and phosphorylated neurofilament heavy (pNfH) as prognostic and potential pharmacodynamic biomarkers relevant to amyotrophic lateral sclerosis (ALS) therapy development.

Methods

In this prospective, multicenter, longitudinal observational study of patients with ALS (n = 229), primary lateral sclerosis (n = 20), and progressive muscular atrophy (n = 11), biological specimens were collected, processed, and stored according to strict standard operating procedures (SOPs). Neurofilament assays were performed in a blinded manner by independent contract research organizations.

Results

For serum NfL and pNfH measured using the Simoa assay, there were no missing data (i

SUBMITTER: Benatar M 

PROVIDER: S-EPMC7371380 | biostudies-literature | 2020 Jul

REPOSITORIES: biostudies-literature