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.
Preview |
Text
Gillmore_Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy_AAM.pdf Download (255kB) | Preview |
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.
| Type: | Article |
|---|---|
| Title: | Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.17925/HI.2023.17.1.27 |
| Publisher version: | https://doi.org/10.17925/HI.2023.17.1.27 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions. |
| Keywords: | Amyloidosis, cardiomyopathy, patisiran, polyneuropathy, safety, transthyretin, transthyretin-mediated amyloidosis |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10175053 |
Loading...
Loading...Loading...Loading...Archive Staff Only
 |
View Item |
