{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T00:59:15Z","timestamp":1790384355778,"version":"4.1.0"},"reference-count":96,"publisher":"Springer Science and Business Media LLC","issue":"5","license":[{"start":{"date-parts":[[2026,7,15]],"date-time":"2026-07-15T00:00:00Z","timestamp":1784073600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2026,7,15]],"date-time":"2026-07-15T00:00:00Z","timestamp":1784073600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"funder":[{"DOI":"10.13039\/501100003478","name":"Ministry of Health, Labour and Welfare","doi-asserted-by":"publisher","award":["26FC0201"],"award-info":[{"award-number":["26FC0201"]}],"id":[{"id":"10.13039\/501100003478","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Neurol Ther"],"published-print":{"date-parts":[[2026,10]]},"DOI":"10.1007\/s40120-026-00994-0","type":"journal-article","created":{"date-parts":[[2026,7,15]],"date-time":"2026-07-15T17:41:26Z","timestamp":1784137286000},"page":"2341-2358","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Disease-Modifying Therapies for Hereditary Transthyretin Amyloidosis with Polyneuropathy: Current Status and Future Perspectives"],"prefix":"10.1007","volume":"15","author":[{"given":"Toshihiro","family":"Ide","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masaaki","family":"Yoshikawa","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4612-3183","authenticated-orcid":false,"given":"Haruki","family":"Koike","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2026,7,15]]},"reference":[{"key":"994_CR1","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. https:\/\/doi.org\/10.1038\/s41582-019-0210-4.","journal-title":"Nat Rev Neurol"},{"key":"994_CR2","doi-asserted-by":"publisher","first-page":"317","DOI":"10.1007\/s40120-020-00210-7","volume":"9","author":"H Koike","year":"2020","unstructured":"Koike H, Katsuno M. Transthyretin amyloidosis: update on the clinical spectrum, pathogenesis, and disease-modifying therapies. Neurol Ther. 2020;9:317\u201333. https:\/\/doi.org\/10.1007\/s40120-020-00210-7.","journal-title":"Neurol Ther"},{"key":"994_CR3","doi-asserted-by":"publisher","first-page":"256","DOI":"10.1210\/endo-63-2-256","volume":"63","author":"SH Ingbar","year":"1958","unstructured":"Ingbar SH. Pre-albumin: a thyroxinebinding protein of human plasma. Endocrinology. 1958;63:256\u20139. https:\/\/doi.org\/10.1210\/endo-63-2-256.","journal-title":"Endocrinology"},{"key":"994_CR4","doi-asserted-by":"publisher","first-page":"2025","DOI":"10.1172\/JCI105889","volume":"47","author":"M Kanai","year":"1968","unstructured":"Kanai M, Raz A, Goodman DS. Retinol-binding protein: the transport protein for vitamin A in human plasma. J Clin Invest. 1968;47:2025\u201344. https:\/\/doi.org\/10.1172\/JCI105889.","journal-title":"J Clin Invest"},{"key":"994_CR5","doi-asserted-by":"publisher","first-page":"73","DOI":"10.1016\/j.cell.2005.01.018","volume":"121","author":"Y Sekijima","year":"2005","unstructured":"Sekijima Y, Wiseman RL, Matteson J, et al. The biological and chemical basis for tissue-selective amyloid disease. Cell. 2005;121:73\u201385. https:\/\/doi.org\/10.1016\/j.cell.2005.01.018.","journal-title":"Cell"},{"key":"994_CR6","doi-asserted-by":"publisher","first-page":"2220","DOI":"10.1212\/WNL.0000000000003362","volume":"87","author":"H Koike","year":"2016","unstructured":"Koike H, Ikeda S, Takahashi M, et al. Schwann cell and endothelial cell damage in transthyretin familial amyloid polyneuropathy. Neurology. 2016;87:2220\u20139. https:\/\/doi.org\/10.1212\/WNL.0000000000003362.","journal-title":"Neurology"},{"key":"994_CR7","doi-asserted-by":"publisher","first-page":"1025","DOI":"10.1212\/wnl.34.8.1025","volume":"34","author":"G Said","year":"1984","unstructured":"Said G, Ropert A, Faux N. Length-dependent degeneration of fibers in Portuguese amyloid polyneuropathy: a clinicopathologic study. Neurology. 1984;34:1025\u201332. https:\/\/doi.org\/10.1212\/wnl.34.8.1025.","journal-title":"Neurology"},{"key":"994_CR8","doi-asserted-by":"publisher","first-page":"323","DOI":"10.1186\/s13023-023-02910-3","volume":"18","author":"Y Ando","year":"2023","unstructured":"Ando Y, Waddington-Cruz M, Sekijima Y, et al. Optimal practices for the management of hereditary transthyretin amyloidosis: real-world experience from Japan, Brazil, and Portugal. Orphanet J Rare Dis. 2023;18:323. https:\/\/doi.org\/10.1186\/s13023-023-02910-3.","journal-title":"Orphanet J Rare Dis"},{"key":"994_CR9","doi-asserted-by":"publisher","first-page":"1771","DOI":"10.1001\/archneur.59.11.1771","volume":"59","author":"H Koike","year":"2002","unstructured":"Koike H, Misu K, Ikeda S, et al. Type I (transthyretin Met30) familial amyloid polyneuropathy in Japan: early- vs late-onset form. Arch Neurol. 2002;59:1771\u20136. https:\/\/doi.org\/10.1001\/archneur.59.11.1771.","journal-title":"Arch Neurol"},{"key":"994_CR10","doi-asserted-by":"publisher","first-page":"53","DOI":"10.3109\/13506129.2011.565524","volume":"18","author":"H Koike","year":"2011","unstructured":"Koike H, Hashimoto R, Tomita M, et al. Diagnosis of sporadic transthyretin Val30Met familial amyloid polyneuropathy: a practical analysis. Amyloid. 2011;18:53\u201362. https:\/\/doi.org\/10.3109\/13506129.2011.565524.","journal-title":"Amyloid"},{"key":"994_CR11","doi-asserted-by":"publisher","first-page":"1086","DOI":"10.1016\/S1474-4422(11)70246-0","volume":"10","author":"V Plant\uff83\uff69-Bordeneuve","year":"2011","unstructured":"Plant\u00e9-Bordeneuve V, Said G. Familial amyloid polyneuropathy. Lancet Neurol. 2011;10:1086\u201397. https:\/\/doi.org\/10.1016\/S1474-4422(11)70246-0.","journal-title":"Lancet Neurol"},{"key":"994_CR12","doi-asserted-by":"publisher","first-page":"152","DOI":"10.1136\/jnnp-2011-301299","volume":"83","author":"H Koike","year":"2012","unstructured":"Koike H, Tanaka F, Hashimoto R, et al. Natural history of transthyretin Val30Met familial amyloid polyneuropathy: analysis of late-onset cases from non-endemic areas. J Neurol Neurosurg Psychiatry. 2012;83:152\u20138. https:\/\/doi.org\/10.1136\/jnnp-2011-301299.","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"994_CR13","doi-asserted-by":"publisher","first-page":"2597","DOI":"10.1111\/j.1600-6143.2007.01969.x","volume":"7","author":"S Yamamoto","year":"2007","unstructured":"Yamamoto S, Wilczek HE, Nowak G, et al. Liver transplantation for familial amyloidotic polyneuropathy (FAP): a single-center experience over 16 years. Am J Transplant. 2007;7:2597\u2013604. https:\/\/doi.org\/10.1111\/j.1600-6143.2007.01969.x.","journal-title":"Am J Transplant"},{"key":"994_CR14","doi-asserted-by":"publisher","first-page":"637","DOI":"10.1212\/WNL.0b013e318248df18","volume":"78","author":"T Yamashita","year":"2012","unstructured":"Yamashita T, Ando Y, Okamoto S, et al. Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy. Neurology. 2012;78:637\u201343. https:\/\/doi.org\/10.1212\/WNL.0b013e318248df18.","journal-title":"Neurology"},{"key":"994_CR15","doi-asserted-by":"publisher","first-page":"235","DOI":"10.1111\/j.1600-6143.2006.01585.x","volume":"7","author":"M Yazaki","year":"2007","unstructured":"Yazaki M, Mitsuhashi S, Tokuda T, et al. Progressive wild-type transthyretin deposition after liver transplantation preferentially occurs onto myocardium in FAP patients. Am J Transplant. 2007;7:235\u201342. https:\/\/doi.org\/10.1111\/j.1600-6143.2006.01585.x.","journal-title":"Am J Transplant"},{"key":"994_CR16","doi-asserted-by":"publisher","first-page":"785","DOI":"10.1212\/WNL.0b013e3182661eb1","volume":"79","author":"T Coelho","year":"2012","unstructured":"Coelho T, Maia LF, Martins da Silva A, et al. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology. 2012;79:785\u201392. https:\/\/doi.org\/10.1212\/WNL.0b013e3182661eb1.","journal-title":"Neurology"},{"key":"994_CR17","doi-asserted-by":"publisher","first-page":"2658","DOI":"10.1001\/jama.2013.283815","volume":"310","author":"JL Berk","year":"2013","unstructured":"Berk JL, Suhr OB, Obici L, et al. Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial. JAMA. 2013;310:2658\u201367. https:\/\/doi.org\/10.1001\/jama.2013.283815.","journal-title":"JAMA"},{"key":"994_CR18","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. https:\/\/doi.org\/10.1056\/NEJMoa1716153.","journal-title":"N Engl J Med"},{"key":"994_CR19","doi-asserted-by":"publisher","first-page":"22","DOI":"10.1056\/NEJMoa1716793","volume":"379","author":"MD Benson","year":"2018","unstructured":"Benson MD, Waddington-Cruz M, Berk JL, et al. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med. 2018;379:22\u201331. https:\/\/doi.org\/10.1056\/NEJMoa1716793.","journal-title":"N Engl J Med"},{"key":"994_CR20","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. https:\/\/doi.org\/10.1080\/13506129.2022.2091985.","journal-title":"Amyloid"},{"key":"994_CR21","doi-asserted-by":"publisher","first-page":"1448","DOI":"10.1001\/jama.2023.18688","volume":"330","author":"T Coelho","year":"2023","unstructured":"Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy. JAMA. 2023;330:1448\u201358. https:\/\/doi.org\/10.1001\/jama.2023.18688.","journal-title":"JAMA"},{"key":"994_CR22","doi-asserted-by":"publisher","first-page":"230","DOI":"10.1002\/ana.26845","volume":"95","author":"M Ueda","year":"2024","unstructured":"Ueda M, Misumi Y, Nomura T, et al. Disease-modifying drugs extend survival in hereditary transthyretin amyloid polyneuropathy. Ann Neurol. 2024;95:230\u20136. https:\/\/doi.org\/10.1002\/ana.26845.","journal-title":"Ann Neurol"},{"key":"994_CR23","doi-asserted-by":"publisher","first-page":"493","DOI":"10.1056\/NEJMoa2107454","volume":"385","author":"JD Gillmore","year":"2021","unstructured":"Gillmore JD, Gane E, Taubel J, et al. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N Engl J Med. 2021;385:493\u2013502. https:\/\/doi.org\/10.1056\/NEJMoa2107454.","journal-title":"N Engl J Med"},{"key":"994_CR24","doi-asserted-by":"publisher","first-page":"441","DOI":"10.1016\/j.neures.2006.08.014","volume":"56","author":"K Tojo","year":"2006","unstructured":"Tojo K, Sekijima Y, Kelly JW, Ikeda S. Diflunisal stabilizes familial amyloid polyneuropathy-associated transthyretin variant tetramers in serum against dissociation required for amyloidogenesis. Neurosci Res. 2006;56:441\u20139. https:\/\/doi.org\/10.1016\/j.neures.2006.08.014.","journal-title":"Neurosci Res"},{"key":"994_CR25","doi-asserted-by":"publisher","first-page":"9629","DOI":"10.1073\/pnas.1121005109","volume":"109","author":"CE Bulawa","year":"2012","unstructured":"Bulawa CE, Connelly S, Devit M, et al. Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade. Proc Natl Acad Sci U S A. 2012;109:9629\u201334. https:\/\/doi.org\/10.1073\/pnas.1121005109.","journal-title":"Proc Natl Acad Sci U S A"},{"key":"994_CR26","doi-asserted-by":"publisher","first-page":"103","DOI":"10.1080\/13506129.2019.1627312","volume":"26","author":"I Concei\u00e7\u00e3o","year":"2019","unstructured":"Concei\u00e7\u00e3o I, Coelho T, Rapezzi C, et al. Assessment of patients with hereditary transthyretin amyloidosis - understanding the impact of management and disease progression. Amyloid. 2019;26:103\u201311. https:\/\/doi.org\/10.1080\/13506129.2019.1627312.","journal-title":"Amyloid."},{"key":"994_CR27","doi-asserted-by":"publisher","first-page":"1061","DOI":"10.1016\/S1474-4422(23)00334-4","volume":"22","author":"D Adams","year":"2023","unstructured":"Adams D, Sekijima Y, Concei\u00e7\u00e3o I, et al. Hereditary transthyretin amyloid neuropathies: advances in pathophysiology, biomarkers, and treatment. Lancet Neurol. 2023;22:1061\u201374. https:\/\/doi.org\/10.1016\/S1474-4422(23)00334-4.","journal-title":"Lancet Neurol."},{"key":"994_CR28","doi-asserted-by":"publisher","first-page":"2802","DOI":"10.1007\/s00415-013-7051-7","volume":"260","author":"T Coelho","year":"2013","unstructured":"Coelho T, Maia LF, da Silva AM, et al. Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. J Neurol. 2013;260:2802\u201314. https:\/\/doi.org\/10.1007\/s00415-013-7051-7.","journal-title":"J Neurol"},{"key":"994_CR29","doi-asserted-by":"publisher","first-page":"178","DOI":"10.1080\/13506129.2016.1207163","volume":"23","author":"M Waddington Cruz","year":"2016","unstructured":"Waddington Cruz M, Amass L, Keohane D, Schwartz J, Li H, Gundapaneni B. Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy. Amyloid. 2016;23:178\u201383. https:\/\/doi.org\/10.1080\/13506129.2016.1207163.","journal-title":"Amyloid"},{"key":"994_CR30","doi-asserted-by":"publisher","first-page":"87","DOI":"10.1007\/s40120-018-0097-9","volume":"7","author":"R Mundayat","year":"2018","unstructured":"Mundayat R, Stewart M, Alvir J, et al. Positive effectiveness of tafamidis in delaying disease progression in transthyretin familial amyloid polyneuropathy up to 2 years: an analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS). Neurol Ther. 2018;7:87\u2013101. https:\/\/doi.org\/10.1007\/s40120-018-0097-9.","journal-title":"Neurol Ther"},{"key":"994_CR31","doi-asserted-by":"publisher","first-page":"1007","DOI":"10.1056\/NEJMoa1805689","volume":"379","author":"MS Maurer","year":"2018","unstructured":"Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018;379:1007\u201316. https:\/\/doi.org\/10.1056\/NEJMoa1805689.","journal-title":"N Engl J Med"},{"key":"994_CR32","doi-asserted-by":"publisher","first-page":"1036","DOI":"10.1136\/jnnp-2014-308724","volume":"86","author":"Y Sekijima","year":"2015","unstructured":"Sekijima Y. Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments. J Neurol Neurosurg Psychiatry. 2015;86:1036\u201343. https:\/\/doi.org\/10.1136\/jnnp-2014-308724.","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"994_CR33","doi-asserted-by":"publisher","first-page":"9992","DOI":"10.1073\/pnas.1300761110","volume":"110","author":"SC Penchala","year":"2013","unstructured":"Penchala SC, Connelly S, Wang Y, et al. AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin. Proc Natl Acad Sci U S A. 2013;110:9992\u20137. https:\/\/doi.org\/10.1073\/pnas.1300761110.","journal-title":"Proc Natl Acad Sci U S A"},{"key":"994_CR34","doi-asserted-by":"publisher","first-page":"285","DOI":"10.1016\/j.jacc.2019.03.012","volume":"74","author":"DP Judge","year":"2019","unstructured":"Judge DP, Heitner SB, Falk RH, et al. Transthyretin stabilization by AG10 in symptomatic transthyretin amyloid cardiomyopathy. J Am Coll Cardiol. 2019;74:285\u201395. https:\/\/doi.org\/10.1016\/j.jacc.2019.03.012.","journal-title":"J Am Coll Cardiol"},{"key":"994_CR35","doi-asserted-by":"publisher","first-page":"132","DOI":"10.1056\/NEJMoa2305434","volume":"390","author":"JD Gillmore","year":"2024","unstructured":"Gillmore JD, Judge DP, Cappelli F, et al. Efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy. N Engl J Med. 2024;390:132\u201342. https:\/\/doi.org\/10.1056\/NEJMoa2305434.","journal-title":"N Engl J Med"},{"issue":"Suppl 1","key":"994_CR36","doi-asserted-by":"publisher","first-page":"43","DOI":"10.3109\/13506129.2012.673140","volume":"19","author":"EJ Ackermann","year":"2012","unstructured":"Ackermann EJ, Guo S, Booten S, et al. Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy. Amyloid. 2012;19(Suppl 1):43\u20134. https:\/\/doi.org\/10.3109\/13506129.2012.673140.","journal-title":"Amyloid"},{"key":"994_CR37","doi-asserted-by":"publisher","first-page":"819","DOI":"10.1056\/NEJMoa1208760","volume":"369","author":"T Coelho","year":"2013","unstructured":"Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013;369:819\u201329. https:\/\/doi.org\/10.1056\/NEJMoa1208760.","journal-title":"N Engl J Med"},{"key":"994_CR38","doi-asserted-by":"publisher","first-page":"49","DOI":"10.1016\/S1474-4422(20)30368-9","volume":"20","author":"D Adams","year":"2021","unstructured":"Adams D, Polydefkis M, Gonz\u00e1lez-Duarte A, et al. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Lancet Neurol. 2021;20:49\u201359. https:\/\/doi.org\/10.1016\/S1474-4422(20)30368-9.","journal-title":"Lancet Neurol."},{"key":"994_CR39","doi-asserted-by":"publisher","first-page":"16958","DOI":"10.1021\/ja505986a","volume":"136","author":"JK Nair","year":"2014","unstructured":"Nair JK, Willoughby JL, Chan A, et al. Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing. J Am Chem Soc. 2014;136:16958\u201361. https:\/\/doi.org\/10.1021\/ja505986a.","journal-title":"J Am Chem Soc"},{"key":"994_CR40","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. https:\/\/doi.org\/10.1007\/s40120-023-00522-4.","journal-title":"Neurol Ther"},{"key":"994_CR41","doi-asserted-by":"publisher","first-page":"393","DOI":"10.1080\/17460441.2024.2306843","volume":"19","author":"V Plant\u00e9-Bordeneuve","year":"2024","unstructured":"Plant\u00e9-Bordeneuve V, Perrain V. Vutrisiran: a new drug in the treatment landscape of hereditary transthyretin amyloid polyneuropathy. Expert Opin Drug Discov. 2024;19:393\u2013402. https:\/\/doi.org\/10.1080\/17460441.2024.2306843.","journal-title":"Expert Opin Drug Discov"},{"key":"994_CR42","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. https:\/\/doi.org\/10.1056\/NEJMoa2409134.","journal-title":"N Engl J Med"},{"key":"994_CR43","doi-asserted-by":"publisher","first-page":"609","DOI":"10.1002\/mus.20503","volume":"33","author":"MD Benson","year":"2006","unstructured":"Benson MD, Kluve-Beckerman B, Zeldenrust SR, et al. Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides. Muscle Nerve. 2006;33:609\u201318. https:\/\/doi.org\/10.1002\/mus.20503.","journal-title":"Muscle Nerve"},{"key":"994_CR44","doi-asserted-by":"publisher","first-page":"1374","DOI":"10.1111\/ene.14285","volume":"27","author":"TH Brannagan","year":"2020","unstructured":"Brannagan TH, Wang AK, Coelho T, et al. Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial. Eur J Neurol. 2020;27:1374\u201381. https:\/\/doi.org\/10.1111\/ene.14285.","journal-title":"Eur J Neurol"},{"key":"994_CR45","doi-asserted-by":"publisher","first-page":"52","DOI":"10.1080\/13506129.2019.1685487","volume":"27","author":"NR Dasgupta","year":"2020","unstructured":"Dasgupta NR, Rissing SM, Smith J, Jung J, Benson MD. Inotersen therapy of transthyretin amyloid cardiomyopathy. Amyloid. 2020;27:52\u20138. https:\/\/doi.org\/10.1080\/13506129.2019.1685487.","journal-title":"Amyloid"},{"key":"994_CR46","doi-asserted-by":"publisher","first-page":"127","DOI":"10.1007\/s40259-023-00577-7","volume":"37","author":"A Ioannou","year":"2023","unstructured":"Ioannou A, Fontana M, Gillmore JD. RNA targeting and gene editing strategies for transthyretin amyloidosis. BioDrugs. 2023;37:127\u201342. https:\/\/doi.org\/10.1007\/s40259-023-00577-7.","journal-title":"BioDrugs"},{"key":"994_CR47","doi-asserted-by":"publisher","first-page":"816","DOI":"10.1126\/science.1225829","volume":"337","author":"M Jinek","year":"2012","unstructured":"Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012;337:816\u201321. https:\/\/doi.org\/10.1126\/science.1225829.","journal-title":"Science"},{"key":"994_CR48","doi-asserted-by":"publisher","first-page":"655","DOI":"10.1038\/s41569-022-00683-z","volume":"19","author":"A Aimo","year":"2022","unstructured":"Aimo A, Castiglione V, Rapezzi C, et al. RNA-targeting and gene editing therapies for transthyretin amyloidosis. Nat Rev Cardiol. 2022;19:655\u201367. https:\/\/doi.org\/10.1038\/s41569-022-00683-z.","journal-title":"Nat Rev Cardiol"},{"key":"994_CR49","doi-asserted-by":"publisher","first-page":"2231","DOI":"10.1056\/NEJMoa2412309","volume":"391","author":"M Fontana","year":"2024","unstructured":"Fontana M, Solomon SD, Kachadourian J, et al. CRISPR-Cas9 gene editing with nexiguran ziclumeran for ATTR cardiomyopathy. N Engl J Med. 2024;391:2231\u201341. https:\/\/doi.org\/10.1056\/NEJMoa2412309.","journal-title":"N Engl J Med"},{"key":"994_CR50","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. https:\/\/doi.org\/10.1001\/jama.2024.0442.","journal-title":"JAMA"},{"key":"994_CR51","doi-asserted-by":"publisher","first-page":"591","DOI":"10.1007\/s40259-022-00550-w","volume":"36","author":"M Nuvolone","year":"2022","unstructured":"Nuvolone M, Nevone A, Merlini G. Targeting amyloid fibrils by passive immunotherapy in systemic amyloidosis. BioDrugs. 2022;36:591\u2013608. https:\/\/doi.org\/10.1007\/s40259-022-00550-w.","journal-title":"BioDrugs"},{"key":"994_CR52","doi-asserted-by":"publisher","first-page":"239","DOI":"10.1056\/NEJMoa2303765","volume":"389","author":"P Garcia-Pavia","year":"2023","unstructured":"Garcia-Pavia P, Aus dem Siepen F, Donal E, et al. Phase 1 trial of antibody NI006 for depletion of cardiac transthyretin amyloid. N Engl J Med. 2023;389:239\u201350. https:\/\/doi.org\/10.1056\/NEJMoa2303765.","journal-title":"N Engl J Med"},{"key":"994_CR53","doi-asserted-by":"publisher","first-page":"14","DOI":"10.1080\/13506129.2024.2420809","volume":"32","author":"OB Suhr","year":"2025","unstructured":"Suhr OB, Grogan M, Silva AMD, et al. PRX004 in variant amyloid transthyretin (ATTRv) amyloidosis: results of a phase 1, open-label, dose-escalation study. Amyloid. 2025;32:14\u201321. https:\/\/doi.org\/10.1080\/13506129.2024.2420809.","journal-title":"Amyloid"},{"key":"994_CR54","doi-asserted-by":"publisher","first-page":"214","DOI":"10.1161\/CIRCULATIONAHA.125.077304","volume":"153","author":"M Fontana","year":"2026","unstructured":"Fontana M, Garc\u00eda-Pav\u00eda P, Grogan M, et al. Coramitug, a humanized monoclonal antibody for the treatment of transthyretin amyloid cardiomyopathy: a phase 2, randomized, multicenter, double-blind. Placebo-Controll Trial Circ. 2026;153:214\u201325. https:\/\/doi.org\/10.1161\/CIRCULATIONAHA.125.077304.","journal-title":"Placebo-Controll Trial Circ"},{"key":"994_CR55","doi-asserted-by":"publisher","first-page":"30","DOI":"10.17925\/HI.2024.18.1.5","volume":"18","author":"A Ioannou","year":"2024","unstructured":"Ioannou A. Evolution of disease-modifying therapy for transthyretin cardiac amyloidosis. Heart Int. 2024;18:30\u20137. https:\/\/doi.org\/10.17925\/HI.2024.18.1.5.","journal-title":"Heart Int"},{"key":"994_CR56","doi-asserted-by":"publisher","first-page":"18","DOI":"10.1038\/s44386-025-00015-4","volume":"2","author":"JS Wall","year":"2025","unstructured":"Wall JS, Williams AD, Balachandran M, et al. The antibody-peptide fusion protein, AT-02, is an effective opsonin with pan-amyloid reactivity. npj Drug Discov. 2025;2:18. https:\/\/doi.org\/10.1038\/s44386-025-00015-4.","journal-title":"npj Drug Discov"},{"key":"994_CR57","doi-asserted-by":"publisher","first-page":"74","DOI":"10.1186\/1479-5876-8-74","volume":"8","author":"I Cardoso","year":"2010","unstructured":"Cardoso I, Martins D, Ribeiro T, Merlini G, Saraiva MJ. Synergy of combined doxycycline\/TUDCA treatment in lowering transthyretin deposition and associated biomarkers: studies in FAP mouse models. J Transl Med. 2010;8:74. https:\/\/doi.org\/10.1186\/1479-5876-8-74.","journal-title":"J Transl Med"},{"issue":"Suppl 1","key":"994_CR58","doi-asserted-by":"publisher","first-page":"34","DOI":"10.3109\/13506129.2012.678508","volume":"19","author":"L Obici","year":"2012","unstructured":"Obici L, Cortese A, Lozza A, et al. Doxycycline plus tauroursodeoxycholic acid for transthyretin amyloidosis: a phase II study. Amyloid. 2012;19(Suppl 1):34\u20136. https:\/\/doi.org\/10.3109\/13506129.2012.678508.","journal-title":"Amyloid"},{"key":"994_CR59","doi-asserted-by":"publisher","DOI":"10.3389\/fnmol.2020.592644","volume":"13","author":"F Bezerra","year":"2020","unstructured":"Bezerra F, Saraiva MJ, Almeida MR. Modulation of the mechanisms driving transthyretin amyloidosis. Front Mol Neurosci. 2020;13:592644. https:\/\/doi.org\/10.3389\/fnmol.2020.592644.","journal-title":"Front Mol Neurosci"},{"key":"994_CR60","doi-asserted-by":"publisher","first-page":"11259","DOI":"10.1074\/jbc.RA119.007851","volume":"294","author":"M Ueda","year":"2019","unstructured":"Ueda M, Okada M, Mizuguchi M, et al. A cell-based high-throughput screening method to directly examine transthyretin amyloid fibril formation at neutral pH. J Biol Chem. 2019;294:11259\u201375. https:\/\/doi.org\/10.1074\/jbc.RA119.007851.","journal-title":"J Biol Chem"},{"key":"994_CR61","doi-asserted-by":"publisher","first-page":"114170","DOI":"10.1016\/j.isci.2025.114170","volume":"29","author":"A Kagami","year":"2025","unstructured":"Kagami A, Hashimoto N, Sasaki R, et al. Glycosidic scaffold bearing multiple galloyl moieties from pomegranate disrupts transthyretin amyloids. iScience. 2025;29:114170. https:\/\/doi.org\/10.1016\/j.isci.2025.114170.","journal-title":"iScience"},{"key":"994_CR62","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1055\/s-0044-1793936","volume":"83","author":"LF Pinto","year":"2025","unstructured":"Pinto LF, Pinto MV, Accioli P, et al. Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil. Arq Neuropsiquiatr. 2025;83:1\u20136. https:\/\/doi.org\/10.1055\/s-0044-1793936.","journal-title":"Arq Neuropsiquiatr"},{"key":"994_CR63","doi-asserted-by":"publisher","first-page":"446","DOI":"10.1001\/jamacardio.2026.0819","volume":"11","author":"P Soman","year":"2026","unstructured":"Soman P, Cuddy SAM, Gillmore JD, et al. Long-term durability of acoramidis efficacy in transthyretin amyloid cardiomyopathy: open-label extension of the ATTRibute-CM randomized clinical trial. JAMA Cardiol. 2026;11:446\u201354. https:\/\/doi.org\/10.1001\/jamacardio.2026.0819.","journal-title":"JAMA Cardiol"},{"key":"994_CR64","doi-asserted-by":"publisher","first-page":"194","DOI":"10.1080\/13506129.2017.1357545","volume":"24","author":"FA Barroso","year":"2017","unstructured":"Barroso FA, Judge DP, Ebede B, et al. Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid. 2017;24:194\u2013204. https:\/\/doi.org\/10.1080\/13506129.2017.1357545.","journal-title":"Amyloid"},{"key":"994_CR65","doi-asserted-by":"publisher","first-page":"51","DOI":"10.2147\/DHPS.S338577","volume":"15","author":"C Falc\u00e3o de Campos","year":"2017","unstructured":"Falc\u00e3o de Campos C, Concei\u00e7\u00e3o I. Updated evaluation of the safety, efficacy and tolerability of tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathy. Drug Healthc Patient Saf. 2023;15:51\u201362. https:\/\/doi.org\/10.2147\/DHPS.S338577.","journal-title":"Drug Healthc Patient Saf"},{"key":"994_CR66","doi-asserted-by":"publisher","first-page":"517","DOI":"10.1007\/s10741-021-10143-4","volume":"27","author":"M Ibrahim","year":"2022","unstructured":"Ibrahim M, Saint Croix GR, Lacy S, et al. The use of diflunisal for transthyretin cardiac amyloidosis: a review. Heart Fail Rev. 2022;27:517\u201324. https:\/\/doi.org\/10.1007\/s10741-021-10143-4.","journal-title":"Heart Fail Rev"},{"key":"994_CR67","doi-asserted-by":"publisher","first-page":"333","DOI":"10.1007\/s40119-025-00423-7","volume":"14","author":"ET Powers","year":"2025","unstructured":"Powers ET, Amass L, Baylor L, Fern\u00e1ndez-Arias I, Riley S, Kelly JW. Transthyretin kinetic stabilizers for ATTR amyloidosis: a narrative review of mechanisms and therapeutic benefits. Cardiol Ther. 2025;14:333\u201350. https:\/\/doi.org\/10.1007\/s40119-025-00423-7.","journal-title":"Cardiol Ther."},{"key":"994_CR68","doi-asserted-by":"publisher","first-page":"1214","DOI":"10.1161\/CIRCULATIONAHA.119.045093","volume":"141","author":"DS Kazi","year":"2020","unstructured":"Kazi DS, Bellows BK, Baron SJ, et al. Cost-effectiveness of tafamidis therapy for transthyretin amyloid cardiomyopathy. Circulation. 2020;141:1214\u201324. https:\/\/doi.org\/10.1161\/CIRCULATIONAHA.119.045093.","journal-title":"Circulation"},{"key":"994_CR69","doi-asserted-by":"publisher","first-page":"141","DOI":"10.1016\/j.bsheal.2025.05.003","volume":"7","author":"X Li","year":"2025","unstructured":"Li X, Gao Y, Zhang Z, et al. Biosafety considerations triggered by genome-editing technologies. Biosaf Health. 2025;7:141\u201351. https:\/\/doi.org\/10.1016\/j.bsheal.2025.05.003.","journal-title":"Biosaf Health"},{"key":"994_CR70","doi-asserted-by":"publisher","first-page":"89","DOI":"10.1080\/13506129.2019.1701429","volume":"27","author":"M In\u00eas","year":"2020","unstructured":"In\u00eas M, Coelho T, Concei\u00e7\u00e3o I, Landeiro F, de Carvalho M, Costa J. Societal costs and burden of hereditary transthyretin amyloidosis polyneuropathy. Amyloid. 2020;27:89\u201396. https:\/\/doi.org\/10.1080\/13506129.2019.1701429.","journal-title":"Amyloid."},{"key":"994_CR71","doi-asserted-by":"publisher","first-page":"473","DOI":"10.1007\/s40120-020-00194-4","volume":"9","author":"SR Reddy","year":"2020","unstructured":"Reddy SR, Chang E, Tarbox MH, et al. The clinical and economic burden of newly diagnosed hereditary transthyretin (ATTRv) amyloidosis: a retrospective analysis of claims data. Neurol Ther. 2020;9:473\u201382. https:\/\/doi.org\/10.1007\/s40120-020-00194-4.","journal-title":"Neurol Ther"},{"key":"994_CR72","doi-asserted-by":"publisher","first-page":"967","DOI":"10.1080\/14737167.2021.1900738","volume":"21","author":"L Galan","year":"2021","unstructured":"Galan L, Gonzalez-Moreno J, Mart\u00ednez-Sesmero JM, et al. Estimating the annual economic burden for the management of patients with transthyretin amyloid polyneuropathy in Spain. Expert Rev Pharmacoecon Outcomes Res. 2021;21:967\u201373. https:\/\/doi.org\/10.1080\/14737167.2021.1900738.","journal-title":"Expert Rev Pharmacoecon Outcomes Res"},{"key":"994_CR73","doi-asserted-by":"publisher","first-page":"422","DOI":"10.1093\/ehjqcco\/qcaf152","volume":"12","author":"A Aimo","year":"2026","unstructured":"Aimo A, Lorenzoni V, Castiglione V, Vergaro G, Emdin M. Cost-effectiveness of vutrisiran, tafamidis, and their combination in amyloid transthyretin cardiomyopathy. Eur Heart J Qual Care Clin Outcomes. 2026;12:422\u20134. https:\/\/doi.org\/10.1093\/ehjqcco\/qcaf152.","journal-title":"Eur Heart J Qual Care Clin Outcomes"},{"key":"994_CR74","doi-asserted-by":"publisher","first-page":"169","DOI":"10.1002\/mus.26515","volume":"60","author":"A Yarlas","year":"2019","unstructured":"Yarlas A, Gertz MA, Dasgupta NR, et al. Burden of hereditary transthyretin amyloidosis on quality of life. Muscle Nerve. 2019;60:169\u201375. https:\/\/doi.org\/10.1002\/mus.26515.","journal-title":"Muscle Nerve"},{"key":"994_CR75","doi-asserted-by":"publisher","first-page":"175","DOI":"10.1080\/13506129.2022.2043270","volume":"29","author":"FA Barroso","year":"2022","unstructured":"Barroso FA, Coelho T, Dispenzieri A, et al. Characteristics of patients with autonomic dysfunction in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid. 2022;29:175\u201383. https:\/\/doi.org\/10.1080\/13506129.2022.2043270.","journal-title":"Amyloid"},{"key":"994_CR76","doi-asserted-by":"publisher","first-page":"349","DOI":"10.1007\/s40120-018-0106-z","volume":"7","author":"M Stewart","year":"2018","unstructured":"Stewart M, Shaffer S, Murphy B, et al. Characterizing the high disease burden of transthyretin amyloidosis for patients and caregivers. Neurol Ther. 2018;7:349\u201364. https:\/\/doi.org\/10.1007\/s40120-018-0106-z.","journal-title":"Neurol Ther"},{"key":"994_CR77","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1186\/s13023-022-02601-5","volume":"18","author":"S Acaster","year":"2023","unstructured":"Acaster S, Lo SH, Nestler-Parr S. A survey exploring caregiver burden and health-related quality of life in hereditary transthyretin amyloidosis. Orphanet J Rare Dis. 2023;18:17. https:\/\/doi.org\/10.1186\/s13023-022-02601-5.","journal-title":"Orphanet J Rare Dis"},{"key":"994_CR78","doi-asserted-by":"publisher","first-page":"129","DOI":"10.1212\/01.wnl.0000132966.36437.12","volume":"63","author":"H Koike","year":"2004","unstructured":"Koike H, Misu K, Sugiura M, et al. Pathology of early- vs late-onset TTR Met30 familial amyloid polyneuropathy. Neurology. 2004;63:129\u201338. https:\/\/doi.org\/10.1212\/01.wnl.0000132966.36437.12.","journal-title":"Neurology"},{"key":"994_CR79","doi-asserted-by":"publisher","first-page":"95","DOI":"10.1186\/s13023-025-03589-4","volume":"20","author":"Y P\u00e9r\u00e9on","year":"2025","unstructured":"P\u00e9r\u00e9on Y, Adams D, Camdessanch\u00e9 JP, et al. Diagnosis of hereditary transthyretin amyloidosis in patients with suspected chronic inflammatory demyelinating polyneuropathy unresponsive to intravenous immunoglobulins: results of a retrospective study. Orphanet J Rare Dis. 2025;20:95. https:\/\/doi.org\/10.1186\/s13023-025-03589-4.","journal-title":"Orphanet J Rare Dis."},{"key":"994_CR80","doi-asserted-by":"publisher","DOI":"10.1080\/13506129.2026.2613126","author":"T Ide","year":"2026","unstructured":"Ide T, Yoshikawa M, Suzuyama K, et al. Significance of aberrant nerve conduction in hereditary transthyretin amyloidosis. Amyloid. 2026;33:196-204.\u00a0https:\/\/doi.org\/10.1080\/13506129.2026.2613126.","journal-title":"Amyloid"},{"key":"994_CR81","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16065","volume":"31","author":"V Di Stefano","year":"2024","unstructured":"Di Stefano V, Lupica A, Alonge P, et al. Genetic screening for hereditary transthyretin amyloidosis with polyneuropathy in western Sicily: two years of experience in a neurological clinic. Eur J Neurol. 2024;31:e16065. https:\/\/doi.org\/10.1111\/ene.16065.","journal-title":"Eur J Neurol"},{"key":"994_CR82","doi-asserted-by":"publisher","first-page":"929","DOI":"10.1007\/s10072-024-07793-1","volume":"46","author":"B Antunes","year":"2024","unstructured":"Antunes B, Concei\u00e7\u00e3o I, Falc\u00e3o de Campos C, de Carvalho M. Diagnostic delay in patients with sporadic hereditary transthyretin-mediated amyloidosis. Neurol Sci. 2025;46:929\u2013934. https:\/\/doi.org\/10.1007\/s10072-024-07793-1.","journal-title":"Neurol Sci"},{"key":"994_CR83","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2020.116813","volume":"414","author":"M Ueda","year":"2020","unstructured":"Ueda M, Sekijima Y, Koike H, et al. Monitoring of asymptomatic family members at risk of hereditary transthyretin amyloidosis for early intervention with disease-modifying therapies. J Neurol Sci. 2020;414:116813. https:\/\/doi.org\/10.1016\/j.jns.2020.116813.","journal-title":"J Neurol Sci"},{"key":"994_CR84","doi-asserted-by":"publisher","first-page":"236","DOI":"10.1186\/s13023-022-02359-w","volume":"17","author":"A Dispenzieri","year":"2022","unstructured":"Dispenzieri A, Coelho T, Concei\u00e7\u00e3o I, et al. Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update. Orphanet J Rare Dis. 2022;17:236. https:\/\/doi.org\/10.1186\/s13023-022-02359-w.","journal-title":"Orphanet J Rare Dis."},{"key":"994_CR85","doi-asserted-by":"publisher","first-page":"445","DOI":"10.1080\/13506129.2023.2229484","volume":"30","author":"T Coelho","year":"2023","unstructured":"Coelho T, Dispenzieri A, Grogan M, et al. Patients with transthyretin amyloidosis enrolled in THAOS between 2018 and 2021 continue to experience substantial diagnostic delay. Amyloid. 2023;30:445\u20138. https:\/\/doi.org\/10.1080\/13506129.2023.2229484.","journal-title":"Amyloid"},{"key":"994_CR86","doi-asserted-by":"publisher","first-page":"890","DOI":"10.1016\/s0006-291x(85)80027-9","volume":"127","author":"PW Dickson","year":"1985","unstructured":"Dickson PW, Aldred AR, Marley PD, Tu GF, Howlett GJ, Schreiber G. High prealbumin and transferrin mRNA levels in the choroid plexus of rat brain. Biochem Biophys Res Commun. 1985;127:890\u20135. https:\/\/doi.org\/10.1016\/s0006-291x(85)80027-9.","journal-title":"Biochem Biophys Res Commun"},{"key":"994_CR87","doi-asserted-by":"publisher","first-page":"11793","DOI":"10.1016\/S0021-9258(17)39100-7","volume":"260","author":"DR Soprano","year":"1985","unstructured":"Soprano DR, Herbert J, Soprano KJ, Schon EA, Goodman DS. Demonstration of transthyretin mRNA in the brain and other extrahepatic tissues in the rat. J Biol Chem. 1985;260:11793\u20138. https:\/\/doi.org\/10.1016\/S0021-9258(17)39100-7.","journal-title":"J Biol Chem"},{"key":"994_CR88","doi-asserted-by":"publisher","first-page":"773","DOI":"10.1212\/WNL.0000000000003001","volume":"87","author":"Y Sekijima","year":"2016","unstructured":"Sekijima Y, Yazaki M, Oguchi K, et al. Cerebral amyloid angiopathy in posttransplant patients with hereditary ATTR amyloidosis. Neurology. 2016;87:773\u201381. https:\/\/doi.org\/10.1212\/WNL.0000000000003001.","journal-title":"Neurology"},{"key":"994_CR89","doi-asserted-by":"publisher","first-page":"117","DOI":"10.1080\/13506129.2024.2435573","volume":"32","author":"Y Sekijima","year":"2025","unstructured":"Sekijima Y, Sousa L. Pathogenesis, manifestations, diagnosis, and management of CNS complications in hereditary ATTR amyloidosis. Amyloid. 2025;32:117\u201328. https:\/\/doi.org\/10.1080\/13506129.2024.2435573.","journal-title":"Amyloid"},{"key":"994_CR90","doi-asserted-by":"publisher","first-page":"931","DOI":"10.7326\/0003-4819-135-10-200111200-00026","volume":"135","author":"E Ando","year":"2001","unstructured":"Ando E, Ando Y, Haraoka K. Ocular amyloid involvement after liver transplantation for polyneuropathy. Ann Intern Med. 2001;135:931\u20132. https:\/\/doi.org\/10.7326\/0003-4819-135-10-200111200-00026.","journal-title":"Ann Intern Med"},{"key":"994_CR91","doi-asserted-by":"publisher","first-page":"120","DOI":"10.1080\/13506129.2018.1479249","volume":"25","author":"C Monteiro","year":"2018","unstructured":"Monteiro C, Martins da Silva A, Ferreira N, et al. Cerebrospinal fluid and vitreous body exposure to orally administered tafamidis in hereditary ATTRV30M (p.TTRV50M) amyloidosis patients. Amyloid. 2018;25:120\u20138. https:\/\/doi.org\/10.1080\/13506129.2018.1479249.","journal-title":"Amyloid"},{"key":"994_CR92","doi-asserted-by":"publisher","DOI":"10.1111\/jns.70088","volume":"30","author":"VJW Zhang","year":"2025","unstructured":"Zhang VJW, Ritter LM, Hoskote C, et al. Impact of high-dose tafamidis on hereditary ATTR (ATTRv) amyloidosis with central nervous system involvement: two case reports with clinical, radiological and cerebrospinal fluid follow up. J Peripher Nerv Syst. 2025;30:e70088. https:\/\/doi.org\/10.1111\/jns.70088.","journal-title":"J Peripher Nerv Syst"},{"key":"994_CR93","doi-asserted-by":"publisher","first-page":"2543","DOI":"10.2174\/1570159X21666230623094710","volume":"21","author":"C Cambieri","year":"2023","unstructured":"Cambieri C, Marenco M, Colasanti T, et al. Does patisiran reduce ocular transthyretin synthesis? a pilot study of two cases. Curr Neuropharmacol. 2023;21:2543\u20139. https:\/\/doi.org\/10.2174\/1570159X21666230623094710.","journal-title":"Curr Neuropharmacol"},{"key":"994_CR94","doi-asserted-by":"publisher","first-page":"350","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. https:\/\/doi.org\/10.1186\/s13023-023-02962-5.","journal-title":"Orphanet J Rare Dis"},{"key":"994_CR95","doi-asserted-by":"publisher","first-page":"34","DOI":"10.1186\/s13023-019-1000-1","volume":"14","author":"M Waddington-Cruz","year":"2019","unstructured":"Waddington-Cruz M, Schmidt H, Botteman MF, et al. Epidemiological and clinical characteristics of symptomatic hereditary transthyretin amyloid polyneuropathy: a global case series. Orphanet J Rare Dis. 2019;14:34. https:\/\/doi.org\/10.1186\/s13023-019-1000-1.","journal-title":"Orphanet J Rare Dis"},{"key":"994_CR96","doi-asserted-by":"publisher","first-page":"e412","DOI":"10.1212\/WNL.0000000000011090","volume":"96","author":"S Ticau","year":"2021","unstructured":"Ticau S, Sridharan GV, Tsour S, et al. Neurofilament light chain as a biomarker of hereditary transthyretin-mediated amyloidosis. Neurology. 2021;96:e412\u201322. https:\/\/doi.org\/10.1212\/WNL.0000000000011090.","journal-title":"Neurology"}],"container-title":["Neurology and Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40120-026-00994-0.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40120-026-00994-0","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40120-026-00994-0.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T00:03:29Z","timestamp":1790381009000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40120-026-00994-0"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,7,15]]},"references-count":96,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2026,10]]}},"alternative-id":["994"],"URL":"https:\/\/doi.org\/10.1007\/s40120-026-00994-0","relation":{},"ISSN":["2193-8253","2193-6536"],"issn-type":[{"value":"2193-8253","type":"print"},{"value":"2193-6536","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,7,15]]},"assertion":[{"value":"23 May 2026","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"18 June 2026","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"15 July 2026","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Haruki Koike is a member of the Editorial Board of\n                      Neurology and Therapy\n                      . Haruki Koike was not involved in the selection of peer reviewers for the manuscript nor any subsequent editorial decisions. Toshihiro Ide and Masaaki Yoshikawa have nothing to disclose.","order":1,"name":"Ethics","label":"Conflicts of Interest","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"This article is based on previously conducted studies and does not contain any new studies with human participants or animals.","order":2,"name":"Ethics","label":"Ethical Approval","group":{"name":"EthicsHeading","label":"Declarations"}}]}}