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- Title
Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy.
- Authors
Ioannou, Adam; Fontana, Marianna; Gillmore, Julian D.
- 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.
- Subjects
AMYLOID; BIOMARKERS; PERIPHERAL neuropathy; AMYLOIDOSIS; CLINICAL trials; NEUROLOGICAL disorders; CARDIOMYOPATHIES; THYROXINE; SMALL interfering RNA; TREATMENT effectiveness; VITAMIN A; POLYNEUROPATHIES; CARDIAC output; QUALITY of life; CARRIER proteins; PATIENT safety
- Publication
Heart International, 2023, Vol 17, Issue 1, p27
- ISSN
1826-1868
- Publication type
Article
- DOI
10.17925/HI.2023.17.1.27