{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T23:19:27Z","timestamp":1787008767026,"version":"build-2736575974"},"reference-count":15,"publisher":"Oxford University Press (OUP)","issue":"Supplement_5","license":[{"start":{"date-parts":[[2026,3,11]],"date-time":"2026-03-11T00:00:00Z","timestamp":1773187200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,5,6]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Transthyretin amyloidosis (ATTR) is a systemic disease characterized by the deposition of misfolded transthyretin (TTR) fibrils. It most commonly presents as cardiomyopathy (ATTR-CM), particularly in elderly patients with the wild-type form, whereas hereditary variants may manifest with polyneuropathy (ATTRv-PN) and\/or cardiac involvement. The widespread adoption of non-invasive diagnostic pathways has increased disease recognition and facilitated the early initiation of disease-modifying therapies. Stabilizers limit dissociation of the TTR tetramer and the formation of new amyloid fibrils. Tafamidis demonstrated a benefit on mortality and cardiovascular hospitalizations in the ATTR-ACT trial and remains the treatment supported by the most robust evidence, with greater efficacy when initiated at earlier disease stages. Acoramidis, a next-generation stabilizer, improved a hierarchical composite end-point in the ATTRibute-CM trial and has been approved for ATTR-CM treatment, too. Gene-silencing therapies (small interfering RNA and antisense oligonucleotides) reduce hepatic TTR synthesis and circulating protein levels. Patisiran is approved for ATTRv-PN and, in the APOLLO-B trial, showed a functional benefit in ATTR-CM, although it has not been approved for this indication in the United States. Vutrisiran, a subcutaneously administered siRNA given every 12 weeks, reduced mortality and recurrent cardiovascular events in the HELIOS-B trial and has been approved for ATTR-CM treatment. This review summarizes the rationale and principal clinical evidence supporting tetramer stabilizers and gene-silencing therapies, which are now central to the management of ATTR-CM.<\/jats:p>","DOI":"10.1093\/eurheartjsupp\/suag034","type":"journal-article","created":{"date-parts":[[2026,3,7]],"date-time":"2026-03-07T12:45:22Z","timestamp":1772887522000},"page":"v100-v105","source":"Crossref","is-referenced-by-count":1,"title":["Therapy of amyloidosis: stabilizers and silencers"],"prefix":"10.1093","volume":"28","author":[{"given":"Alberto","family":"Aimo","sequence":"first","affiliation":[{"name":"Health Science Interdisciplinary Center, Scuola Superiore Sant\u2019Anna , via Cardinale Maffi 27, 56126 Pisa , Italy"},{"name":"Cardiology and Cardiovascular Medicine Division, Fondazione Toscana Gabriele Monasterio , via Moruzzi 1, 56124 Pisa, 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