SOD1 and amyotrophic lateral sclerosis: Patients (n = 21) with a family history of ALS and carrying a documented heterozygous mutation in SOD1 were enrolled into four cohorts of eight (some were re-enrolled) and treated with a single, small volume (0.25 ml) intrathecal administration of SOD1 ASO or placebo over 11.5 h via a lumbar intraspinal catheter.