{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,13]],"date-time":"2026-07-13T13:06:07Z","timestamp":1783947967950,"version":"3.55.0"},"reference-count":42,"publisher":"Elsevier BV","issue":"9","license":[{"start":{"date-parts":[[2024,9,1]],"date-time":"2024-09-01T00:00:00Z","timestamp":1725148800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,9,1]],"date-time":"2024-09-01T00:00:00Z","timestamp":1725148800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,3,13]],"date-time":"2024-03-13T00:00:00Z","timestamp":1710288000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006400","name":"Alnylam Pharmaceuticals Inc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006400","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.jp","clinicalkey.es","clinicalkey.com.au","clinicalkey.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of the Formosan Medical Association"],"published-print":{"date-parts":[[2024,9]]},"DOI":"10.1016\/j.jfma.2024.03.008","type":"journal-article","created":{"date-parts":[[2024,3,27]],"date-time":"2024-03-27T22:01:31Z","timestamp":1711576891000},"page":"975-984","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":4,"title":["Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study"],"prefix":"10.1016","volume":"123","author":[{"given":"Kon-Ping","family":"Lin","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chih-Chao","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yi-Chung","family":"Lee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ming-Jen","family":"Lee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Vest","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianne T.","family":"Sweetser","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7655-8145","authenticated-orcid":false,"given":"Matthew T.","family":"White","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1248-616X","authenticated-orcid":false,"given":"Prajakta","family":"Badri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sung-Tsang","family":"Hsieh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6499-5789","authenticated-orcid":false,"given":"Chi-Chao","family":"Chao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib1","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1185\/03007995.2012.754348","article-title":"THAOS \u2013 the Transthyretin Amyloidosis Outcomes Survey: initial report on clinical manifestations in patients with hereditary and wild-type transthyretin amyloidosis","volume":"29","author":"Coelho","year":"2013","journal-title":"Curr Med Res Opin"},{"key":"10.1016\/j.jfma.2024.03.008_bib2","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1186\/1750-1172-8-31","article-title":"Guideline of transthyretin-related hereditary amyloidosis for clinicians","volume":"8","author":"Ando","year":"2013","journal-title":"Orphanet J Rare Dis"},{"issue":"2","key":"10.1016\/j.jfma.2024.03.008_bib3","first-page":"150","article-title":"Familial amyloid polyneuropathy","volume":"56","author":"Cakar","year":"2019","journal-title":"Noro Psikiyatr Ars"},{"issue":"Suppl 1","key":"10.1016\/j.jfma.2024.03.008_bib4","doi-asserted-by":"crossref","first-page":"S14","DOI":"10.1097\/WCO.0000000000000289","article-title":"First European consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy","volume":"29","author":"Adams","year":"2016","journal-title":"Curr Opin Neurol"},{"issue":"8","key":"10.1016\/j.jfma.2024.03.008_bib5","doi-asserted-by":"crossref","first-page":"625","DOI":"10.3109\/07853890.2015.1068949","article-title":"Evolving landscape in the management of transthyretin amyloidosis","volume":"47","author":"Hawkins","year":"2015","journal-title":"Ann Med"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib6","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1056\/NEJMoa1716153","article-title":"Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis","volume":"379","author":"Adams","year":"2018","journal-title":"N Engl J Med"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib7","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1056\/NEJMoa1716793","article-title":"Inotersen treatment for patients with hereditary transthyretin amyloidosis","volume":"379","author":"Benson","year":"2018","journal-title":"N Engl J Med"},{"issue":"2","key":"10.1016\/j.jfma.2024.03.008_bib8","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1007\/s10741-014-9462-7","article-title":"Natural history and therapy of TTR-cardiac amyloidosis: Emerging disease-modifying therapies from organ transplantation to stabilizer and silencer drugs","volume":"20","author":"Casta\u00f1o","year":"2015","journal-title":"Heart Fail Rev"},{"issue":"2","key":"10.1016\/j.jfma.2024.03.008_bib9","doi-asserted-by":"crossref","first-page":"123","DOI":"10.3109\/13506129.2015.1019610","article-title":"Hereditary ATTR amyloidosis: a single-institution experience with 266 patients","volume":"22","author":"Swiecicki","year":"2015","journal-title":"Amyloid"},{"issue":"9","key":"10.1016\/j.jfma.2024.03.008_bib10","doi-asserted-by":"crossref","first-page":"1120","DOI":"10.1093\/eurheartj\/ehr383","article-title":"Cardiac phenotype and clinical outcome of familial amyloid polyneuropathy associated with transthyretin alanine 60 variant","volume":"33","author":"Sattianayagam","year":"2012","journal-title":"Eur Heart J"},{"issue":"5","key":"10.1016\/j.jfma.2024.03.008_bib11","doi-asserted-by":"crossref","first-page":"428","DOI":"10.1016\/S0025-6196(12)60388-7","article-title":"Familial amyloidosis: a study of 52 North American-born patients examined during a 30-year period","volume":"67","author":"Gertz","year":"1992","journal-title":"Mayo Clin Proc"},{"issue":"5","key":"10.1016\/j.jfma.2024.03.008_bib12","doi-asserted-by":"crossref","first-page":"913","DOI":"10.1002\/acn3.778","article-title":"Clinical and genetic profiles of hereditary transthyretin amyloidosis in Taiwan","volume":"6","author":"Chao","year":"2019","journal-title":"Ann Clin Transl Neurol"},{"key":"10.1016\/j.jfma.2024.03.008_bib13","unstructured":"Health Promotion Administration, Ministry of Health and Welfare. Statistical report of rare disease confirmed cases in Taiwan. Accessed July 2023. https:\/\/www.hpa.gov.tw\/EngPages\/Detail.aspx?nodeid=4715&pid=17404."},{"issue":"21","key":"10.1016\/j.jfma.2024.03.008_bib14","doi-asserted-by":"crossref","first-page":"2616","DOI":"10.1097\/CM9.0000000000001094","article-title":"Clinical and genetic features of transthyretin-related familial amyloid polyneuropathy in China","volume":"133","author":"Liu","year":"2020","journal-title":"Chin Med J (Engl)"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib15","doi-asserted-by":"crossref","first-page":"206","DOI":"10.1186\/s12883-021-02243-3","article-title":"Ala97Ser transthyretin amyloidosis-associated polyneuropathy, clinical and neurophysiological profiles in a Thai cohort","volume":"21","author":"Pasutharnchat","year":"2021","journal-title":"BMC Neurol"},{"issue":"Suppl 1","key":"10.1016\/j.jfma.2024.03.008_bib16","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1080\/13506129.2019.1582479","article-title":"Ala97Ser mutation is common among ethnic Chinese Malaysians with transthyretin familial amyloid polyneuropathy","volume":"26","author":"Low","year":"2019","journal-title":"Amyloid"},{"issue":"3","key":"10.1016\/j.jfma.2024.03.008_bib17","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1080\/13506129.2017.1357545","article-title":"Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years","volume":"24","author":"Barroso","year":"2017","journal-title":"Amyloid"},{"key":"10.1016\/j.jfma.2024.03.008_bib18","series-title":"Summary of product characteristics: Vyndaqel 20 mg soft capsules","year":"2018"},{"issue":"6","key":"10.1016\/j.jfma.2024.03.008_bib19","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1111\/j.1751-7133.2012.00303.x","article-title":"Diflunisal for ATTR cardiac amyloidosis","volume":"18","author":"Castano","year":"2012","journal-title":"Congest Heart Fail"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib20","doi-asserted-by":"crossref","first-page":"5","DOI":"10.2217\/nmt-2018-0033","article-title":"Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis","volume":"9","author":"Kristen","year":"2019","journal-title":"Neurodegener Dis Manag"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib21","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1016\/S1474-4422(20)30368-9","article-title":"Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study","volume":"20","author":"Adams","year":"2021","journal-title":"Lancet Neurol"},{"key":"10.1016\/j.jfma.2024.03.008_bib22","series-title":"Global open-label extension: 24-month data of patisiran in patients with hATTR amyloidosis. Peripheral Nerve Society (PNS) Congress","author":"Adams","year":"2020"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib23","doi-asserted-by":"crossref","first-page":"178","DOI":"10.1186\/s12883-017-0957-4","article-title":"Phenotypic expressions of hereditary transthyretin ala97Ser related amyloidosis (ATTR) in Taiwanese","volume":"17","author":"Hsu","year":"2017","journal-title":"BMC Neurol"},{"issue":"6","key":"10.1016\/j.jfma.2024.03.008_bib24","doi-asserted-by":"crossref","first-page":"532","DOI":"10.1212\/WNL.0b013e3181ec7fda","article-title":"Clinical presentations and skin denervation in amyloid neuropathy due to transthyretin Ala97Ser","volume":"75","author":"Yang","year":"2010","journal-title":"Neurology"},{"issue":"2","key":"10.1016\/j.jfma.2024.03.008_bib25","doi-asserted-by":"crossref","first-page":"272","DOI":"10.1002\/ana.24438","article-title":"Sudomotor innervation in transthyretin amyloid neuropathy: pathology and functional correlates","volume":"78","author":"Chao","year":"2015","journal-title":"Ann Neurol"},{"issue":"11","key":"10.1016\/j.jfma.2024.03.008_bib26","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0141935","article-title":"The temporal profiles of changes in nerve excitability indices in familial amyloid polyneuropathy","volume":"10","author":"Lai","year":"2015","journal-title":"PLoS One"},{"key":"10.1016\/j.jfma.2024.03.008_bib27","article-title":"Genetic analysis of hereditary transthyretin Ala97Ser related amyloidosis","author":"Hsu","year":"2018","journal-title":"J Vis Exp [serial online]"},{"key":"10.1016\/j.jfma.2024.03.008_bib28","article-title":"Unique phenotypes with corresponding pathology in late-onset hereditary transthyretin amyloidosis of A97S vs. V30M","volume":"13","author":"Hsueh","year":"2021","journal-title":"Front Aging Neurosci"},{"issue":"5","key":"10.1016\/j.jfma.2024.03.008_bib29","doi-asserted-by":"crossref","first-page":"363","DOI":"10.1093\/jnen\/nlac022","article-title":"Distinct patterns of amyloid pathology in autopsies of transthyretin S77Y and A97S amyloidosis: significance of symptomatology and cell biology","volume":"81","author":"Chang","year":"2022","journal-title":"J Neuropathol Exp Neurol"},{"issue":"3","key":"10.1016\/j.jfma.2024.03.008_bib30","doi-asserted-by":"crossref","first-page":"693","DOI":"10.1016\/j.jfma.2019.08.027","article-title":"Cardiac manifestations and prognostic implications of hereditary transthyretin amyloidosis associated with transthyretin Ala97Ser","volume":"119","author":"Lai","year":"2020","journal-title":"J Formos Med Assoc"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib31","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1186\/s13023-023-02824-0","article-title":"Tafamidis decreased cardiac amyloidosis deposition in patients with Ala97Ser hereditary transthyretin cardiomyopathy: a 12-month follow-up cohort study","volume":"18","author":"Tsai","year":"2023","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.jfma.2024.03.008_bib32","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1111\/ncn3.12396","article-title":"Patisiran, an RNAi therapeutic for patients with hereditary transthyretin-mediated amyloidosis: sub-analysis in Japanese patients from the APOLLO study","volume":"8","author":"Yamashita","year":"2020","journal-title":"Neurol Clin Neurosci"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib33","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1002\/jcph.1480","article-title":"Patisiran pharmacokinetics, pharmacodynamics, and exposure-response analyses in the phase 3 APOLLO trial in patients with hereditary transthyretin-mediated (hATTR) amyloidosis","volume":"60","author":"Zhang","year":"2020","journal-title":"J Clin Pharmacol"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib34","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1186\/s13023-019-1000-1","article-title":"Epidemiological and clinical characteristics of symptomatic hereditary transthyretin amyloid polyneuropathy: a global case series","volume":"14","author":"Waddington-Cruz","year":"2019","journal-title":"Orphanet J Rare Dis"},{"issue":"6","key":"10.1016\/j.jfma.2024.03.008_bib35","doi-asserted-by":"crossref","first-page":"4911","DOI":"10.1007\/s12035-017-0694-0","article-title":"A missense variant p.Ala117Ser in the transthyretin gene of a Han Chinese family with familial amyloid polyneuropathy","volume":"55","author":"Chen","year":"2018","journal-title":"Mol Neurobiol"},{"issue":"9","key":"10.1016\/j.jfma.2024.03.008_bib36","doi-asserted-by":"crossref","first-page":"1359","DOI":"10.1002\/acn3.51635","article-title":"Cardiomyopathy correlates to nerve damage in p.A117S late-onset transthyretin amyloid polyneuropathy","volume":"9","author":"Lin","year":"2022","journal-title":"Ann Clin Transl Neurol"},{"issue":"1","key":"10.1016\/j.jfma.2024.03.008_bib37","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1080\/13506129.2022.2118043","article-title":"Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial","volume":"30","author":"Quan","year":"2022","journal-title":"Amyloid"},{"issue":"6","key":"10.1016\/j.jfma.2024.03.008_bib38","doi-asserted-by":"crossref","first-page":"2109","DOI":"10.1007\/s00415-019-09688-0","article-title":"Expert consensus recommendations to improve diagnosis of ATTR amyloidosis with polyneuropathy","volume":"268","author":"Adams","year":"2021","journal-title":"J Neurol"},{"issue":"3","key":"10.1016\/j.jfma.2024.03.008_bib39","doi-asserted-by":"crossref","first-page":"982","DOI":"10.1111\/ene.14698","article-title":"Early changes of nerve integrity in preclinical carriers of hereditary transthyretin Ala117Ser amyloidosis with polyneuropathy","volume":"28","author":"Chiang","year":"2021","journal-title":"Eur J Neurol"},{"key":"10.1016\/j.jfma.2024.03.008_bib40","doi-asserted-by":"crossref","DOI":"10.1016\/j.jns.2020.116810","article-title":"Electrophysiological parameters that contribute to the pathogenesis of familial amyloid polyneuropathy caused by transthyretin mutations","volume":"413","author":"Lai","year":"2020","journal-title":"J Neurol Sci"},{"issue":"12","key":"10.1016\/j.jfma.2024.03.008_bib41","doi-asserted-by":"crossref","first-page":"952","DOI":"10.3390\/brainsci10120952","article-title":"Advances in treatment of ATTRv amyloidosis: state of the art and future prospects","volume":"10","author":"Russo","year":"2020","journal-title":"Brain Sci"},{"issue":"2","key":"10.1016\/j.jfma.2024.03.008_bib42","doi-asserted-by":"crossref","first-page":"268","DOI":"10.1007\/s00415-016-8337-3","article-title":"Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study","volume":"264","author":"Plante-Bordeneuve","year":"2017","journal-title":"J Neurol"}],"container-title":["Journal of the Formosan Medical Association"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0929664624001530?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0929664624001530?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,9,8]],"date-time":"2024-09-08T14:26:48Z","timestamp":1725805608000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0929664624001530"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,9]]},"references-count":42,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2024,9]]}},"alternative-id":["S0929664624001530"],"URL":"https:\/\/doi.org\/10.1016\/j.jfma.2024.03.008","relation":{},"ISSN":["0929-6646"],"issn-type":[{"value":"0929-6646","type":"print"}],"subject":[],"published":{"date-parts":[[2024,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study","name":"articletitle","label":"Article Title"},{"value":"Journal of the Formosan Medical Association","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jfma.2024.03.008","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 Formosan Medical Association. Published by Elsevier Taiwan LLC.","name":"copyright","label":"Copyright"}]}}