Daily treatment of patients affected by hereditary ATTR amyloidosis with polyneuropathy (TTR Val30Met mutation) with tafamidis led to circulating TTR tetramer stabilization and, in the efficacy-evaluable cohort of patients, to a significant delay in peripheral neurologic impairment over a 18-month treatment period compared to placebo, even though the coprimary endpoints of the trial were not met (Coelho et al., 2012). This evidence concerns the gene TTR and polyneuropathy.