In NEURO‐TTR, 173 patients with hATTR polyneuropathy were randomly assigned 2:1 to receive subcutaneous inotersen 300 mg once weekly (n = 113; n = 112 treated) or placebo (n = 60) stratified by mutation status, disease stage, and prior stabilizer treatment (Benson et al., 2018). The gene discussed is TTR; the disease is polyneuropathy.