{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,8,2]],"date-time":"2025-08-02T19:03:56Z","timestamp":1754161436664,"version":"3.41.2"},"reference-count":20,"publisher":"Georg Thieme Verlag KG","issue":"07","license":[{"start":{"date-parts":[[2025,6,20]],"date-time":"2025-06-20T00:00:00Z","timestamp":1750377600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Arq Neuropsiquiatr"],"published-print":{"date-parts":[[2025,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Variant transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and cardiomyopathy (ATTRv-CM), formerly known as familial amyloidotic polyneuropathy (FAP), is a severe, progressive disorder caused by mutations in the transthyretin (TTR) gene. Historically, FAP was considered a neglected disease due to its rarity and the limited understanding of its pathophysiology, which led to minimal research funding and few therapeutic options. The present article explores the transformative role of Centro de Paramiloidose Ant\u00f4nio Rodrigues de Mello (CEPARM), established in 1984, in elevating the status of FAP through significant advancements in research and treatment. Although CEPARM was not the sole catalyst for this shift, its contributions in liver transplantation, the development of pharmacological therapies, and holistic patient care have substantially improved the recognition and management of FAP. The article also examines CEPARM's impact on patient care, the ongoing challenges, and ethical considerations within the field.<\/jats:p>","DOI":"10.1055\/s-0045-1809401","type":"journal-article","created":{"date-parts":[[2025,6,20]],"date-time":"2025-06-20T18:32:21Z","timestamp":1750444341000},"page":"001-005","source":"Crossref","is-referenced-by-count":0,"title":["40 years of CEPARM: transforming amyloidosis related to transthyretin from neglect to recognition"],"prefix":"10.1055","volume":"83","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4853-2236","authenticated-orcid":false,"given":"Marcia","family":"Waddington Cruz","sequence":"additional","affiliation":[{"name":"Universidade Federal do Rio de Janeiro, Faculdade de Medicina, Programa de P\u00f3s-Gradua\u00e7\u00e3o em Cl\u00ednica M\u00e9dica, Rio de Janeiro RJ, Brazil."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8889-2573","authenticated-orcid":false,"given":"Marleide","family":"da Mota Gomes","sequence":"additional","affiliation":[{"name":"Universidade Federal do Rio de Janeiro, Instituto de Neurologia, Rio de Janeiro RJ, Brazil."},{"name":"Universidade Federal do Rio de Janeiro, Instituto de Psiquiatria, Laborat\u00f3rio de Hist\u00f3ria da Psiquiatria, Neurologia e Sa\u00fade Mental, Rio de Janeiro RJ, Brazil."}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"194","published-online":{"date-parts":[[2025,6,20]]},"reference":[{"issue":"11","key":"ref1","doi-asserted-by":"publisher","first-page":"1061","DOI":"10.1016\/S1474-4422(23)00334-4","article-title":"Hereditary transthyretin amyloid neuropathies: advances in pathophysiology, biomarkers, and treatment","volume":"22","author":"D Adams","year":"2023","journal-title":"Lancet 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familiar por transtirretina TTR Val30Met e os prim\u00f3rdios do Centro de Estudos de Paramiloidose \/ Principles of the Familial transthyretin amyloidosis TTR Val30Met and the beginning of the Paramyloidosis Center of Antonio Rodrigues de Mello","volume":"47","author":"M M Gomes","year":"2011","journal-title":"Rev Bras Neurol"},{"issue":"02","key":"ref9","first-page":"74","volume":"21","author":"CEPARM","year":"1985","journal-title":"Rev Bras Neurol"},{"issue":"02","key":"ref10","doi-asserted-by":"publisher","first-page":"96","DOI":"10.1590\/0004-282\u2009x\u200920180156","article-title":"Baseline disease characteristics in Brazilian patients enrolled in Transthyretin Amyloidosis Outcome Survey (THAOS)","volume":"77","author":"M W Cruz","year":"2019","journal-title":"Arq Neuropsiquiatr"},{"key":"ref11","doi-asserted-by":"publisher","first-page":"53","DOI":"10.1080\/13506129.2019.1582518","article-title":"Geographic distribution of ATTR cases from CEPARM across the Brazilian territory 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