C9orf72 and amyotrophic lateral sclerosis: With the help of these translational approaches, several clinical trials for patients with ALS were initiated, including a phase I trial of ASOs targeting C9orf72 variants 1 and 3 RNA (BIIB078) (clinicaltrials.gov Identifier: NCT03626012) and a phase I trial of the nucleocytoplasmic transport inhibitor KPT-350 (also called BIIB100) [202] (clinicaltrials.gov identifier: NCT03945279).