2026-04-29T22:51:35Zhttps://repisalud.isciii.es/rest/oai/requestoai:repisalud.isciii.es:20.500.12105/267562025-12-18T12:59:32Zcom_20.500.12105_19604com_20.500.12105_2051col_20.500.12105_19605
Repisalud
author
Gonzalez-Lopez, Esther
author
Maurer, Mathew S
author
Garcia-Pavia, Pablo
funder
Unión Europea. Fondo Europeo de Desarrollo Regional (FEDER/ERDF)
funder
Instituto de Salud Carlos III
funder
Fundación ProCNIC
funder
Ministerio de Ciencia e Innovación. Centro de Excelencia Severo Ochoa (España)
funder
National Institutes of Health (Estados Unidos)
2025-06-17T09:23:54Z
2025-06-17T09:23:54Z
2025-03-13
Eur Heart J. 2025 Mar 13;46(11):999-1013.
European Heart Journal
39791537
https://hdl.handle.net/20.500.12105/26756
Development of specific therapies addressing the underlying diseases' mechanisms constitutes the basis of precision medicine. 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.
eng
Amyloid
Cardiac amyloidosis
Heart failure
Transthyretin
Transthyretin amyloid cardiomyopathy: a paradigm for advancing precision medicine.
research article
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URL
https://repisalud.isciii.es/bitstreams/535906d8-217c-400b-990d-6d63f6395718/download
File
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745b38d36891059e8a3908b240b60291
4231891
application/pdf
Transthyretin amyloid cardiomyopathy_Eur Heart J_2025.pdf