The phase 3 open-label NEURO-TTRansform trial (NCT04136184) met its primary endpoints, demonstrating that eplontersen treatment reduced serum TTR, halted progression of neuropathy impairment, and improved QoL in patients with ATTRv-PN, compared with historical placebo from the NEURO-TTR trial (NCT01737398) of inotersen [19, 20]. This evidence concerns the gene TTR and neuropathy.