Effects of tofersen treatment in patients with <em>SOD1</em>-ALS in a "real-world" setting - a 12-month multicenter cohort study from the German early access program

BACKGROUND: In April 2023, the antisense oligonucleotide tofersen was approved by the U.S. Food and Drug Administration (FDA) for treatment of SOD1-amyotrophic lateral sclerosis (ALS), after a decrease of neurofilament light chain (NfL) levels had been …