eprintid: 10175053
rev_number: 11
eprint_status: archive
userid: 699
dir: disk0/10/17/50/53
datestamp: 2023-08-14 09:59:10
lastmod: 2023-08-14 14:18:41
status_changed: 2023-08-14 09:59:10
type: article
metadata_visibility: show
sword_depositor: 699
creators_name: Ioannou, A
creators_name: Fontana, M
creators_name: Gillmore, JD
title: Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy
ispublished: pub
divisions: UCL
divisions: B02
divisions: C10
divisions: D17
divisions: G90
keywords: Amyloidosis, cardiomyopathy, patisiran, polyneuropathy, safety, transthyretin, transthyretin-mediated amyloidosis
note: This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions.
abstract: Transthyretin (TTR) is a tetrameric protein, synthesized primarily by the liver, that acts as a physiological transport protein for retinol and thyroxine. TTR can misfold into pathogenic amyloid fibrils that deposit in the heart and nerves, causing a life-threatening transthyretin amyloidosis cardiomyopathy (ATTR-CM), and a progressive and debilitating polyneuropathy (ATTR-PN). Recent therapeutic advances have resulted in the development of drugs that reduce TTR production. Patisiran is a small interfering RNA that disrupts the complimentary mRNA and inhibits TTR synthesis, and is the first gene-silencing medication licensed for the treatment of ATTR amyloidosis. After encouraging results following the use of patisiran for the treatment of patients with ATTR-PN, there has been increasing interest in the use of patisiran for the treatment of ATTR-CM. Various studies have demonstrated improvements across a wide range of cardiac biomarkers following treatment with patisiran, and have changed the perception of ATTR-CM from being thought of as a terminal disease process, to now being regarded as a treatable disease. These successes represent a huge milestone and have the potential to revolutionize the landscape of treatment for ATTR-CM. However, the long-term safety of patisiran and how best to monitor cardiac response to treatment remain to be determined.
date: 2023
date_type: published
publisher: Touch Medical Media, Ltd.
official_url: https://doi.org/10.17925/HI.2023.17.1.27
oa_status: green
full_text_type: other
language: eng
primo: open
primo_central: open_green
verified: verified_manual
elements_id: 2038743
doi: 10.17925/HI.2023.17.1.27
medium: Electronic-eCollection
lyricists_name: Gillmore, Julian
lyricists_id: JGILL78
actors_name: Gillmore, Julian
actors_name: Berkeley, Jean
actors_id: JGILL78
actors_id: JBERK64
actors_role: owner
actors_role: impersonator
full_text_status: public
publication: Heart International
volume: 17
number: 1
pagerange: 27-35
event_location: England
citation:        Ioannou, A;    Fontana, M;    Gillmore, JD;      (2023)    Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy.                   Heart International , 17  (1)   pp. 27-35.    10.17925/HI.2023.17.1.27 <https://doi.org/10.17925/HI.2023.17.1.27>.       Green open access   
 
document_url: https://discovery.ucl.ac.uk/id/eprint/10175053/2/Gillmore_Patisiran%20for%20the%20Treatment%20of%20Transthyretin-mediated%20Amyloidosis%20with%20Cardiomyopathy_AAM.pdf