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Transthyretin cardiac amyloidosis (ATTR-CM) is an exemplar of precise therapeutic approach in the field of heart failure and cardiomyopathies. A better understanding of the underlying pathophysiology, more precise data of its epidemiology, and advances in imaging techniques that allow non-invasive diagnosis have fostered the development of new and very effective specific therapies for ATTR-CM. Therapeutic advances have revolutionized the field, transforming a rare, devastating, and untreatable disease into a more common disease with several therapeutic alternatives available. Three main types of therapies (stabilizers, suppressors, and degraders) that act at different points of the amyloidogenic cascade have been developed or are currently under investigation. In this review, the key advances in pathophysiology and epidemiology that have occurred in the last decades along with the different therapeutic alternatives available or under development for ATTR-CM are described, illustrating the role of precision medicine applied to cardiovascular disorders. Pending questions that would need to be answered in upcoming years are also reviewed.<\/jats:p>","DOI":"10.1093\/eurheartj\/ehae811","type":"journal-article","created":{"date-parts":[[2024,11,15]],"date-time":"2024-11-15T07:22:16Z","timestamp":1731655336000},"page":"999-1013","source":"Crossref","is-referenced-by-count":65,"title":["Transthyretin amyloid cardiomyopathy: a paradigm for advancing precision medicine"],"prefix":"10.1093","volume":"46","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4175-1487","authenticated-orcid":false,"given":"Esther","family":"Gonzalez-Lopez","sequence":"first","affiliation":[{"name":"Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA , Manuel de Falla, 1, 28222 Majadahonda, Madrid ,","place":["Spain"]},{"name":"CIBER Cardiovascular, Instituto de Salud Carlos III , Avenida Monforte de Lemos 3-5. Pabell\u00f3n 11. Planta 0. 28029 Madrid ,","place":["Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5400-5008","authenticated-orcid":false,"given":"Mathew S","family":"Maurer","sequence":"additional","affiliation":[{"name":"Division of Cardiology, Department of Medicine, Columbia University Irving Medical Centre , New York, NY ,","place":["USA"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5470-2257","authenticated-orcid":false,"given":"Pablo","family":"Garcia-Pavia","sequence":"additional","affiliation":[{"name":"Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA , Manuel de Falla, 1, 28222 Majadahonda, Madrid ,","place":["Spain"]},{"name":"CIBER Cardiovascular, Instituto de Salud Carlos III , Avenida Monforte de Lemos 3-5. Pabell\u00f3n 11. Planta 0. 28029 Madrid ,","place":["Spain"]},{"name":"Universidad Francisco de Vitoria , M-515; Km 1, 800, 282223 Pozuelo de Alarc\u00f3n, Madrid ,","place":["Spain"]},{"name":"Miocardiopatias Hereditarias, Centro Nacional de Investigaciones Cardiovasculares (CNIC) , Melchor Fern\u00e1ndez Almagro 3, 28029 Madrid ,","place":["Spain"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2025,1,10]]},"reference":[{"key":"2025041112564778300_ehae811-B1","doi-asserted-by":"publisher","first-page":"2517","DOI":"10.1016\/j.jacc.2020.12.071","article-title":"Precision medicine in cardiovascular disease: genetics and impact on phenotypes: JACC Focus Seminar 1\/5","volume":"77","author":"Semsarian","year":"2021 25","journal-title":"J Am Coll Cardiol"},{"key":"2025041112564778300_ehae811-B2","doi-asserted-by":"publisher","first-page":"1042","DOI":"10.1016\/j.rec.2023.06.009","article-title":"Gene therapy for heart failure and cardiomyopathies","volume":"76","author":"Argir\u00f2","year":"2023","journal-title":"Rev Esp Cardiol (Engl Ed)"},{"key":"2025041112564778300_ehae811-B3","doi-asserted-by":"publisher","first-page":"2342","DOI":"10.1002\/ejhf.2532","article-title":"Redefining the epidemiology of cardiac amyloidosis. 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