Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen
Journal
Neurology and therapy
Journal Volume
12
Journal Issue
1
Pages
267
Date Issued
2023-02
Author(s)
Coelho, Teresa
Waddington Cruz, Márcia
Parman, Yeşim
Wixner, Jonas
Weiler, Markus
Barroso, Fabio A
Dasgupta, Noel R
Jung, Shiangtung W
Schneider, Eugene
Viney, Nicholas J
Dyck, P James B
Ando, Yukio
Gillmore, Julian D
Khella, Sami
Gertz, Morie A
Obici, Laura
Berk, John L
Abstract
Hereditary 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.
Subjects
ATTR; Amyloid; Cardiomyopathy; Eplontersen; Polyneuropathy; Transthyretin amyloidosis
Type
journal article