Long-Term Efficacy and Safety of Tofersen in SOD1-ALS Patients

Weekly Neurology Deep Dive - A review of recent impactful publications in the field of Neurology · Mar 3, 2026 · 12m

This paper details the final long-term results of the VALOR study and its open-label extension, which investigated the efficacy of tofersen in adults with SOD1-ALS. Researchers compared patients who began the drug immediately to those who switched from a placebo after six months, finding that earlier treatment led to better outcomes in muscle strength, breathing, and physical function. A critical finding was the sustained reduction of neurofilament light chain levels, a biomarker indicating that the therapy successfully slowed nerve cell degeneration. While the disease is typically…