NEFL and amyotrophic lateral sclerosis: Genetic therapies, such as antisense oligonucleotides (ASOs), offer promise by targeting specific disease-associated genes; for example, the ASO tofersen, which targets SOD1 mutations, has demonstrated target engagement, reduction in SOD1 protein levels, and modulation of neurofilament light chain (NfL) biomarkers in phase 1–2 and phase 3 clinical trials in patients with SOD1-associated ALS [10,11].