SOD1 and amyotrophic lateral sclerosis: Based on the strong functional recovery observed in rodent models and the favourable pharmacokinetics in NHPs, a randomised, double-blind, placebo-controlled Phase I/II study (VALOR; NCT02623699) was initiated to evaluate the tolerability and pharmacokinetics of intrathecal administration of tofersen in familial ALS patients carrying a mutation in the SOD1 gene [70].