The NEURO-TTR [63] (Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis) was a phase III, international, randomised, double-blind, placebo-controlled trial analysing the safety and efficacy of treatment with inotersen in patients with ATTRv with polyneuropathy (ATTR-PN). This evidence concerns the gene TTR and poikiloderma with neutropenia.