TY  - JOUR
IS  - 1
N1  - This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions.
VL  - 17
SP  - 27
JF  - Heart International
A1  - Ioannou, A
A1  - Fontana, M
A1  - Gillmore, JD
UR  - https://doi.org/10.17925/HI.2023.17.1.27
TI  - Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy
AV  - public
Y1  - 2023///
EP  - 35
KW  - Amyloidosis
KW  -  cardiomyopathy
KW  -  patisiran
KW  -  polyneuropathy
KW  -  safety
KW  -  transthyretin
KW  -  transthyretin-mediated amyloidosis
N2  - 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.
ID  - discovery10175053
PB  - Touch Medical Media, Ltd.
ER  -