A natural history comparison Check for updates of SOD1-mutant patients with amyotrophic lateral sclerosis between Chinese and German populations

摘要

Currently,there is no effective treatment for amyotrophic lateral sclerosis(ALS),despite the limited efficacy of riluzole[1]and edaravone[2].SOD1(coding for the Cu/Zn superoxide dismutase)is the second most frequent genetic cause for ALS only after C9orf72 in patients with European ancestry while being the most frequent in Asian ALS populations[3],Multiple therapeutic approaches have targeted SOD1-related ALS,including the antisense oligonucleotide tofersen with promising results in a recent phase I/II trial[4].