{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,2]],"date-time":"2026-05-02T23:40:40Z","timestamp":1777765240228,"version":"3.51.4"},"reference-count":156,"publisher":"MDPI AG","issue":"24","license":[{"start":{"date-parts":[[2022,12,18]],"date-time":"2022-12-18T00:00:00Z","timestamp":1671321600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Italian Ministry of Health","award":["#GR-2018-12368387"],"award-info":[{"award-number":["#GR-2018-12368387"]}]},{"name":"Italian Ministry of Health","award":["#2018-0257"],"award-info":[{"award-number":["#2018-0257"]}]},{"name":"Italian Ministry of Health","award":["C355\/A26819"],"award-info":[{"award-number":["C355\/A26819"]}]},{"name":"CARIPLO Foundation","award":["#GR-2018-12368387"],"award-info":[{"award-number":["#GR-2018-12368387"]}]},{"name":"CARIPLO Foundation","award":["#2018-0257"],"award-info":[{"award-number":["#2018-0257"]}]},{"name":"CARIPLO Foundation","award":["C355\/A26819"],"award-info":[{"award-number":["C355\/A26819"]}]},{"name":"Cancer Research UK","award":["#GR-2018-12368387"],"award-info":[{"award-number":["#GR-2018-12368387"]}]},{"name":"Cancer Research UK","award":["#2018-0257"],"award-info":[{"award-number":["#2018-0257"]}]},{"name":"Cancer Research UK","award":["C355\/A26819"],"award-info":[{"award-number":["C355\/A26819"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>The care of systemic amyloidosis has improved dramatically due to improved awareness, accurate diagnostic tools, the development of powerful prognostic and companion biomarkers, and a continuous flow of innovative drugs, which translated into the blooming of phase 2\/3 interventional studies for light chain (AL) and transthyretin (ATTR) amyloidosis. The unprecedented availability of effective drugs ignited great interest across various medical specialties, particularly among cardiologists who are now recognizing cardiac amyloidosis at an extraordinary pace. In all amyloidosis referral centers, we are observing a substantial increase in the prevalence of wild-type transthyretin (ATTRwt) cardiomyopathy, which is now becoming the most common form of cardiac amyloidosis. This review focuses on the oral drugs that have been recently introduced for the treatment of ATTR cardiac amyloidosis, for their ease of use in the clinic. They include both old repurposed drugs or fit-for-purpose designed compounds which bind and stabilize the TTR tetramer, thus reducing the formation of new amyloid fibrils, such as tafamidis, diflunisal, and acoramidis, as well as fibril disruptors which have the potential to promote the clearance of amyloid deposits, such as doxycycline. The development of novel therapies is based on the advances in the understanding of the molecular events underlying amyloid cardiomyopathy.<\/jats:p>","DOI":"10.3390\/ijms232416145","type":"journal-article","created":{"date-parts":[[2022,12,19]],"date-time":"2022-12-19T05:25:46Z","timestamp":1671427546000},"page":"16145","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Oral Therapy for the Treatment of Transthyretin-Related Amyloid Cardiomyopathy"],"prefix":"10.3390","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8334-1684","authenticated-orcid":false,"given":"Mario","family":"Nuvolone","sequence":"first","affiliation":[{"name":"Department of Molecular Medicine, University of Pavia, 27100 Pavia, Italy"},{"name":"Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, 27100 Pavia, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria","family":"Girelli","sequence":"additional","affiliation":[{"name":"Department of Molecular Medicine, University of Pavia, 27100 Pavia, Italy"},{"name":"Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, 27100 Pavia, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7680-3254","authenticated-orcid":false,"given":"Giampaolo","family":"Merlini","sequence":"additional","affiliation":[{"name":"Department of Molecular Medicine, University of Pavia, 27100 Pavia, Italy"},{"name":"Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, 27100 Pavia, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2022,12,18]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1038\/s41572-018-0034-3","article-title":"Systemic immunoglobulin light chain amyloidosis","volume":"4","author":"Merlini","year":"2018","journal-title":"Nat. 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