Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen

Coelho, TeresaTeresaCoelhoWaddington Cruz, MárciaMárciaWaddington CruzCHI-CHAO CHAOParman, YeşimYeşimParmanWixner, JonasJonasWixnerWeiler, MarkusMarkusWeilerBarroso, Fabio AFabio ABarrosoDasgupta, Noel RNoel RDasguptaJung, Shiangtung WShiangtung WJungSchneider, EugeneEugeneSchneiderViney, Nicholas JNicholas JVineyDyck, P James BP James BDyckAndo, YukioYukioAndoGillmore, Julian DJulian DGillmoreKhella, SamiSamiKhellaGertz, Morie AMorie AGertzObici, LauraLauraObiciBerk, John LJohn LBerk2023-10-242023-10-242023-022193-8253https://scholars.lib.ntu.edu.tw/handle/123456789/636451Hereditary transthyretin (ATTRv) amyloidosis is a rare, severe, progressive, debilitating, and ultimately fatal disease caused by systemic deposition of transthyretin (TTR) amyloid fibrils. ATTRv amyloidosis occurs in both males and females. Eplontersen (ION-682884), a ligand-conjugated antisense oligonucleotide designed to degrade hepatic TTR mRNA, is being evaluated for the treatment of ATTRv amyloidosis with polyneuropathy (ATTRv-PN) in the phase 3, international, multicenter, open-label NEURO-TTRansform study (NCT04136184). To describe the study population of this pivotal trial, here we report the baseline characteristics of patients enrolled in the NEURO-TTRansform study.enATTR; Amyloid; Cardiomyopathy; Eplontersen; Polyneuropathy; Transthyretin amyloidosis[SDGs]SDG3[SDGs]SDG17Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersenjournal article10.1007/s40120-022-00414-z365251402-s2.0-85144143659https://api.elsevier.com/content/abstract/scopus_id/85144143659