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 after 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 supports the notion of tofersen having disease-modifying activity.