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Article

Underlying Immune Disorder May Predispose Some Transthyretin Amyloidosis Subjects to Inotersen-Mediated Thrombocytopenia.

Nucleic acid therapeutics - 1 Apr 2020

Narayanan PadmaKumar, Curtis Brian R, Shen Lijiang, Schneider Eugene, Tami Joseph A, Paz Suzanne, Burel Sebastien A, Tai Li-Jung, Machemer Todd, Kwoh T Jesse, Xia Shuting, Shattil Sanford J, Witztum Joseph L, Engelhardt Jeffery A, Henry Scott P, Monia Brett P, Hughes Steven G

Abstract excerpt

Inotersen, a 2'-O-methoxyethyl (2'-MOE) phosphorothioate antisense oligonucleotide, reduced disease progression and improved quality of life in patients with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) in the NEURO-TTR and NEURO-TTR open-label extension (OLE) trials. However, 300 mg/week inotersen treatment was associated with platelet count reductions in several patients. Mean platelet...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Identifiers

PMID: 32043907
DOI: 10.1089/nat.2019.0829

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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