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

18 , 19 During the initial placebo-controlled phase I-III trial, a significant reduction of neurofilament light chain (NfL) in plasma compared to placebo suggested a disease-modifying effect, 19 although clinical progression parameters such as ALS functional rating scale revised (ALSFRS-R) did not reach statistical significance after six months. 19 However, in the Open Label Extension (OLE) phase of the VALOR study, Miller and colleagues reported after an overall observation period of 52 weeks a lower decrease in ALSFRS-R (difference 3.5 points) in participants, with early-start vs. those with delayed-start of treatment.

also in 111,027 other papers