@article{discovery10175053,
         journal = {Heart International},
       publisher = {Touch Medical Media, Ltd.},
           title = {Patisiran for the Treatment of Transthyretin-mediated Amyloidosis with Cardiomyopathy},
            year = {2023},
          number = {1},
           pages = {27--35},
            note = {This version is the author accepted manuscript. For information on re-use, please refer to the publisher's terms and conditions.},
          volume = {17},
        keywords = {Amyloidosis, cardiomyopathy, patisiran, polyneuropathy, safety, transthyretin, transthyretin-mediated amyloidosis},
          author = {Ioannou, A and Fontana, M and Gillmore, JD},
        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.},
             url = {https://doi.org/10.17925/HI.2023.17.1.27}
}