Safety and Efficacy of Inotersen in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (NEURO-TTR) (N2.001)

To report the safety and efficacy of inotersen, an antisense oligonucleotide inhibitor of transthyretin (TTR) protein production, for the treatment of patients with hereditary TTR amyloidosis (hATTR) in a global, randomized, double-blind, placebo-controlled phase 3 study (NEURO-TTR, NCT01737398).