Neurofilament light chain response during therapy with antisense oligonucleotide Tofersen in SOD1-related ALS - treatment experience in clinical practice.

In this case series, the significant NfL decline following tofersen treatment confirmed its value as response biomarker in an expanded clinical spectrum of SOD1-ALS. Given the previously reported strong correlation between sNfL and ALS progression, the NfL treatment response contributes to the notion of disease-modifying activity of tofersen. This article is protected by copyright. All rights reserved.