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Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis

Authors Mathew V, Wang AK

Received 11 September 2018

Accepted for publication 27 December 2018

Published 6 May 2019 Volume 2019:13 Pages 1515—1525


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 4

Editor who approved publication: Dr Qiongyu Guo

Veena Mathew,1 Annabel K Wang1,2

1Department of Neurology, UCI ALS and Neuromuscular Center, University of California, Irvine, Orange, CA, USA; 2Neurology Section, Tibor Rubin VA Medical Center, Long Beach, CA, USA

Abstract: Hereditary transthyretin amyloidosis is a fatal autosomal dominant disorder characterized by deposition of transthyretin amyloid into the peripheral nervous system, heart, kidney, and gastrointestinal tract. Previous treatments using liver transplantation and small molecule stabilizers were not effective in stopping disease progression. Inotersen, a 2'-O-methyoxyethyl-modified antisense oligonucleotide, which acts by reducing the production of transthyretin, was recently demonstrated to improve disease course and quality of life in early hereditary transthyretin amyloidosis polyneuropathy in a 15-month Phase III study.

Keywords: antisense oligonucleotide, familial amyloid polyneuropathy, Inotersen, mRNA, RNase H

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