{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,5]],"date-time":"2026-08-05T07:50:29Z","timestamp":1785916229137,"version":"3.56.0"},"reference-count":32,"publisher":"Springer Science and Business Media LLC","issue":"3","license":[{"start":{"date-parts":[[2026,3,4]],"date-time":"2026-03-04T00:00:00Z","timestamp":1772582400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2026,3,4]],"date-time":"2026-03-04T00:00:00Z","timestamp":1772582400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"funder":[{"DOI":"10.13039\/100006400","name":"Alnylam Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006400","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Neurol Ther"],"published-print":{"date-parts":[[2026,6]]},"DOI":"10.1007\/s40120-026-00899-y","type":"journal-article","created":{"date-parts":[[2026,3,4]],"date-time":"2026-03-04T14:34:13Z","timestamp":1772634853000},"page":"1065-1080","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Efficacy and Safety of Vutrisiran in Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy: Analysis of the East Asian Subpopulation from HELIOS-A"],"prefix":"10.1007","volume":"15","author":[{"given":"Jeeyoung","family":"Oh","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshiki","family":"Sekijima","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hideki","family":"Mochizuki","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Byoung","family":"Joon Kim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ju-Hong","family":"Min","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masahisa","family":"Katsuno","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cheng","family":"Yin Tan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yi-Chung","family":"Lee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yohei","family":"Misumi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Weizhi","family":"Zhao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy","family":"Chan-Daniels","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chi-Chao","family":"Chao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,3,4]]},"reference":[{"key":"899_CR1","doi-asserted-by":"publisher","first-page":"595","DOI":"10.1007\/s10072-020-04889-2","volume":"43","author":"F Manganelli","year":"2022","unstructured":"Manganelli F, Fabrizi GM, Luigetti M, Mandich P, Mazzeo A, Pareyson D. Hereditary transthyretin amyloidosis overview. Neurol Sci. 2022;43:595\u2013604.","journal-title":"Neurol Sci"},{"key":"899_CR2","doi-asserted-by":"publisher","first-page":"675","DOI":"10.1212\/WNL.0000000000001870","volume":"85","author":"D Adams","year":"2015","unstructured":"Adams D, Coelho T, Obici L, et al. Rapid progression of familial amyloidotic polyneuropathy: a multinational natural history study. Neurology. 2015;85:675\u201382.","journal-title":"Neurology"},{"key":"899_CR3","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCHEARTFAILURE.119.006075","volume":"12","author":"MS Maurer","year":"2019","unstructured":"Maurer MS, Bokhari S, Damy T, et al. Expert consensus recommendations for the suspicion and diagnosis of transthyretin cardiac amyloidosis. Circ Heart Fail. 2019;12:e006075.","journal-title":"Circ Heart Fail"},{"key":"899_CR4","doi-asserted-by":"publisher","first-page":"778","DOI":"10.1001\/jama.2024.0442","volume":"331","author":"FL Ruberg","year":"2024","unstructured":"Ruberg FL, Maurer MS. Cardiac amyloidosis due to transthyretin protein: a review. JAMA. 2024;331:778\u201391.","journal-title":"JAMA"},{"key":"899_CR5","doi-asserted-by":"publisher","first-page":"785","DOI":"10.1007\/s10741-021-10080-2","volume":"27","author":"JN Nativi-Nicolau","year":"2022","unstructured":"Nativi-Nicolau JN, Karam C, Khella S, Maurer MS. Screening for ATTR amyloidosis in the clinic: overlapping disorders, misdiagnosis, and multiorgan awareness. Heart Fail Rev. 2022;27:785\u201393.","journal-title":"Heart Fail Rev"},{"key":"899_CR6","doi-asserted-by":"publisher","first-page":"387","DOI":"10.1038\/s41582-019-0210-4","volume":"15","author":"D Adams","year":"2019","unstructured":"Adams D, Koike H, Slama M, Coelho T. Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease. Nat Rev Neurol. 2019;15:387\u2013404.","journal-title":"Nat Rev Neurol"},{"key":"899_CR7","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-023-02962-5","volume":"18","author":"L Gentile","year":"2023","unstructured":"Gentile L, Coelho T, Dispenzieri A, et al. A 15-year consolidated overview of data in over 6000 patients from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Orphanet J Rare Dis. 2023;18:350.","journal-title":"Orphanet J Rare Dis"},{"key":"899_CR8","doi-asserted-by":"publisher","first-page":"537","DOI":"10.3988\/jcn.2018.14.4.537","volume":"14","author":"K Choi","year":"2018","unstructured":"Choi K, Seok JM, Kim BJ, et al. Characteristics of South Korean patients with hereditary transthyretin amyloidosis. J Clin Neurol. 2018;14:537\u201341.","journal-title":"J Clin Neurol"},{"key":"899_CR9","doi-asserted-by":"publisher","first-page":"642","DOI":"10.1016\/j.nmd.2021.03.008","volume":"31","author":"SC Low","year":"2021","unstructured":"Low SC, Md Sari NA, Tan CY, et al. Hereditary transthyretin amyloidosis in multi-ethnic Malaysians. Neuromuscul Disord. 2021;31:642\u201350.","journal-title":"Neuromuscul Disord"},{"key":"899_CR10","doi-asserted-by":"publisher","first-page":"7","DOI":"10.1080\/13506129.2019.1582479","volume":"26","author":"SC Low","year":"2019","unstructured":"Low SC, Tan CY, Md Sari NA, et al. Ala97Ser mutation is common among ethnic Chinese Malaysians with transthyretin familial amyloid polyneuropathy. Amyloid. 2019;26:7\u20138.","journal-title":"Amyloid"},{"key":"899_CR11","doi-asserted-by":"publisher","DOI":"10.3346\/jkms.2024.39.e163","volume":"39","author":"D Kim","year":"2024","unstructured":"Kim D, Youn JC, Lee HW, et al. Diagnostic pitfall and clinical characteristics of variant versus wild-type transthyretin amyloid cardiomyopathy in Asian population: the Korean Nationwide Cohort Study. J Korean Med Sci. 2024;39:e163.","journal-title":"J Korean Med Sci"},{"key":"899_CR12","doi-asserted-by":"publisher","first-page":"723","DOI":"10.3233\/JND-210656","volume":"8","author":"Z Chen","year":"2021","unstructured":"Chen Z, Koh JS, Saini M, et al. Hereditary transthyretin amyloidosis: clinical and genetic characteristics of a multiracial South-East Asian cohort in Singapore. J Neuromuscul Dis. 2021;8:723\u201333.","journal-title":"J Neuromuscul Dis"},{"key":"899_CR13","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-025-04006-6","volume":"20","author":"T Nomura","year":"2025","unstructured":"Nomura T, Misumi Y, Tasaki M, et al. Genetic and clinical features of hereditary transthyretin amyloidosis: a decade of experience at a Japanese referral center. Orphanet J Rare Dis. 2025;20:474.","journal-title":"Orphanet J Rare Dis"},{"key":"899_CR14","doi-asserted-by":"publisher","first-page":"831","DOI":"10.1002\/acn3.51328","volume":"8","author":"K Du","year":"2021","unstructured":"Du K, Li F, Wang H, et al. Hereditary transthyretin amyloidosis in mainland China: a unicentric retrospective study. Ann Clin Transl Neurol. 2021;8:831\u201341.","journal-title":"Ann Clin Transl Neurol"},{"key":"899_CR15","doi-asserted-by":"publisher","first-page":"1158","DOI":"10.1002\/ana.27203","volume":"97","author":"X Chu","year":"2025","unstructured":"Chu X, Kang J, Xu J, et al. A multicenter study of hereditary transthyretin amyloidosis in China. Ann Neurol. 2025;97:1158\u201367.","journal-title":"Ann Neurol"},{"key":"899_CR16","doi-asserted-by":"publisher","DOI":"10.3389\/fgene.2023.1126836","volume":"14","author":"Z Yongsheng","year":"2023","unstructured":"Yongsheng Z, Chong S, Bingyou L, et al. Prevalence estimation of ATTRv in China based on genetic databases. Front Genet. 2023;14:1126836.","journal-title":"Front Genet"},{"key":"899_CR17","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1080\/13506129.2022.2091985","volume":"30","author":"D Adams","year":"2023","unstructured":"Adams D, Tournev IL, Taylor MS, et al. Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. Amyloid. 2023;30:1\u20139.","journal-title":"Amyloid"},{"key":"899_CR18","doi-asserted-by":"publisher","first-page":"1076","DOI":"10.1016\/j.jacc.2022.11.022","volume":"81","author":"MM Kittleson","year":"2023","unstructured":"Kittleson MM, Ruberg FL, Ambardekar AV, et al. 2023 ACC expert consensus decision pathway on comprehensive multidisciplinary care for the patient\u00a0with cardiac amyloidosis: a report of the American College of Cardiology solution set oversight committee. J Am Coll Cardiol. 2023;81:1076\u2013126.","journal-title":"J Am Coll Cardiol"},{"key":"899_CR19","doi-asserted-by":"publisher","first-page":"273","DOI":"10.1002\/mus.28026","volume":"69","author":"C Karam","year":"2024","unstructured":"Karam C, Mauermann ML, Gonzalez-Duarte A, et al. Diagnosis and treatment of hereditary transthyretin amyloidosis with polyneuropathy in the United States: recommendations from a panel of experts. Muscle Nerve. 2024;69:273\u201387.","journal-title":"Muscle Nerve"},{"key":"899_CR20","doi-asserted-by":"publisher","first-page":"143","DOI":"10.1080\/13506129.2022.2052838","volume":"29","author":"Y Ando","year":"2022","unstructured":"Ando Y, Adams D, Benson MD, et al. Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis. Amyloid. 2022;29:143\u201355.","journal-title":"Amyloid"},{"key":"899_CR21","unstructured":"Alnylam Pharmaceuticals. AMVUTTRA\u00ae (vutrisiran injection), for subcutaneous use [prescribing information]. Cambridge, MA: Alnylam Pharmaceuticals; 2025. https:\/\/www.alnylam.com\/sites\/default\/files\/pdfs\/amvuttra-us-prescribing-information.pdf. Accessed 12 June 2025."},{"key":"899_CR22","doi-asserted-by":"publisher","first-page":"372","DOI":"10.1002\/cpt.1974","volume":"109","author":"BA Habtemariam","year":"2021","unstructured":"Habtemariam BA, Karsten V, Attarwala H, et al. Single-dose pharmacokinetics and pharmacodynamics of transthyretin targeting N-acetylgalactosamine-small interfering ribonucleic acid conjugate, vutrisiran, in healthy subjects. Clin Pharmacol Ther. 2021;109:372\u201382.","journal-title":"Clin Pharmacol Ther"},{"key":"899_CR23","doi-asserted-by":"publisher","first-page":"11","DOI":"10.1056\/NEJMoa1716153","volume":"379","author":"D Adams","year":"2018","unstructured":"Adams D, Gonzalez-Duarte A, O\u2019Riordan WD, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med. 2018;379:11\u201321.","journal-title":"N Engl J Med"},{"key":"899_CR24","doi-asserted-by":"publisher","first-page":"266","DOI":"10.1080\/13506129.2024.2383467","volume":"31","author":"Q Wang","year":"2024","unstructured":"Wang Q, Wang M, Zhu X, et al. Clinical and molecular insights into A97S variants in hereditary transthyretin amyloid polyneuropathy in South China. Amyloid. 2024;31:266\u201374.","journal-title":"Amyloid"},{"key":"899_CR25","doi-asserted-by":"publisher","first-page":"693","DOI":"10.1016\/j.jfma.2019.08.027","volume":"119","author":"HJ Lai","year":"2020","unstructured":"Lai HJ, Huang KC, Liang YC, et al. Cardiac manifestations and prognostic implications of hereditary transthyretin amyloidosis associated with transthyretin Ala97Ser. J Formos Med Assoc. 2020;119:693\u2013700.","journal-title":"J Formos Med Assoc"},{"key":"899_CR26","doi-asserted-by":"publisher","first-page":"913","DOI":"10.1002\/acn3.778","volume":"6","author":"HC Chao","year":"2019","unstructured":"Chao HC, Liao YC, Liu YT, et al. Clinical and genetic profiles of hereditary transthyretin amyloidosis in Taiwan. Ann Clin Transl Neurol. 2019;6:913\u201322.","journal-title":"Ann Clin Transl Neurol"},{"key":"899_CR27","doi-asserted-by":"publisher","first-page":"3449","DOI":"10.1007\/s10072-024-07340-y","volume":"45","author":"SY Tan","year":"2024","unstructured":"Tan SY, Tan CY, Yahya MA, Low SC, Shahrizaila N, Goh KJ. Quantitative muscle ultrasound as a disease biomarker in hereditary transthyretin amyloidosis with polyneuropathy. Neurol Sci. 2024;45:3449\u201359.","journal-title":"Neurol Sci"},{"key":"899_CR28","doi-asserted-by":"publisher","first-page":"33","DOI":"10.1056\/NEJMoa2409134","volume":"392","author":"M Fontana","year":"2025","unstructured":"Fontana M, Berk JL, Gillmore JD, et al. Vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy. N Engl J Med. 2025;392:33\u201344.","journal-title":"N Engl J Med"},{"key":"899_CR29","doi-asserted-by":"publisher","first-page":"397","DOI":"10.1002\/ejhf.3138","volume":"26","author":"P Garcia-Pavia","year":"2024","unstructured":"Garcia-Pavia P, Grogan M, Kale P, et al. Impact of vutrisiran on exploratory cardiac parameters in hereditary transthyretin-mediated amyloidosis with polyneuropathy. Eur J Heart Fail. 2024;26:397\u2013410.","journal-title":"Eur J Heart Fail"},{"key":"899_CR30","doi-asserted-by":"publisher","first-page":"1759","DOI":"10.1007\/s40120-023-00522-4","volume":"12","author":"L Obici","year":"2023","unstructured":"Obici L, Ajroud-Driss S, Lin KP, et al. Impact of vutrisiran on quality of life and physical function in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy. Neurol Ther. 2023;12:1759\u201375.","journal-title":"Neurol Ther"},{"key":"899_CR31","doi-asserted-by":"publisher","first-page":"1599","DOI":"10.2169\/internalmedicine.0091-22","volume":"62","author":"M Matsushima","year":"2023","unstructured":"Matsushima M, Tarisawa M, Nomura T, et al. Practice of hereditary ATTR amyloidosis in non-endemic areas of Japan. Intern Med. 2023;62:1599\u2013602.","journal-title":"Intern Med"},{"key":"899_CR32","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2024.1465747","volume":"15","author":"MA Karimi","year":"2024","unstructured":"Karimi MA, Esmaeilpour Moallem F, Gholami Chahkand MS, Azarm E, Emami Kazemabad MJ, Dadkhah PA. Assessing the effectiveness and safety of patisiran and vutrisiran in ATTRv amyloidosis with polyneuropathy: a systematic review. Front Neurol. 2024;15:1465747.","journal-title":"Front Neurol"}],"container-title":["Neurology and Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40120-026-00899-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40120-026-00899-y","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40120-026-00899-y.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,13]],"date-time":"2026-05-13T14:07:30Z","timestamp":1778681250000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40120-026-00899-y"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,3,4]]},"references-count":32,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,6]]}},"alternative-id":["899"],"URL":"https:\/\/doi.org\/10.1007\/s40120-026-00899-y","relation":{},"ISSN":["2193-8253","2193-6536"],"issn-type":[{"value":"2193-8253","type":"print"},{"value":"2193-6536","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,3,4]]},"assertion":[{"value":"5 December 2025","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"3 February 2026","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"4 March 2026","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Dr. Oh reports nothing to disclose. Dr. Sekijima has received speaker honoraria and research grants from Alnylam Pharmaceuticals and Pfizer, institutional grants from Alnylam Pharmaceuticals and Pfizer, and royalties from Pfizer. Dr. Mochizuki reports nothing to disclose. Dr. Kim reports nothing to disclose. Dr. Min reports nothing to disclose. Dr. Katsuno reports nothing to disclose. Dr. Tan reports nothing to disclose. Dr. Lee reports nothing to disclose. Dr. Misumi declares a research grant from Alnylam Pharmaceuticals. Dr. Zhao and Dr. Chan-Daniels are employees of Alnylam Pharmaceuticals, Inc. and report ownership of equity in Alnylam Pharmaceuticals, Inc. Dr. Chao reports nothing to disclose.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"The study protocol and amendments were approved by relevant Institutional Review Boards or Independent Ethics Committees. This information is detailed in the supplementary material, Table\n                      \n                      . Written informed consent was obtained from each participant. The study was conducted in accordance with all applicable regulatory requirements, the current guidelines of Good Clinical Practice, and principles originating from the Declaration of Helsinki.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}