C9orf72 and amyotrophic lateral sclerosis: For example, polyGP was reduced by the respective ASOs in the WVE-004 and BIIB078 trials in patients with ALS due to C9orf72 hexanucleotide repeat expansions, and mutant huntingtin protein was reduced by the ASO tominersen in patients with HD, but neither was associated with clinical benefits [58,71].