{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,22]],"date-time":"2026-09-22T10:04:07Z","timestamp":1790071447846,"version":"4.0.1"},"reference-count":25,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T00:00:00Z","timestamp":1788480000000},"content-version":"vor","delay-in-days":3,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100013669","name":"Ionis Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013669","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004325","name":"AstraZeneca","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004325","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J Peripheral Nervous Sys"],"published-print":{"date-parts":[[2026,9]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background and Aims<\/jats:title>\n                    <jats:p>In the NEURO\u2010TTRansform clinical trial (NCT04136184), eplontersen improved neuropathy impairment and quality of life (QoL) through Week 66 versus the NEURO\u2010TTR historical placebo in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv\u2010PN). This analysis assessed the impact of baseline ATTRv\u2010PN severity on eplontersen response in patients from NEURO\u2010TTRansform.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      This post hoc analysis grouped patients into tertiles by baseline Neuropathy Impairment Score (NIS): T1 (least baseline impairment: 3.5 to &lt;\u200927.5;\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200967), T2 (27.5 to &lt;\u200955.0;\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200967), and T3 (55.0 to &lt;\u2009127.8;\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200966). Outcomes assessed were neuropathy impairment (modified NIS+7 [mNIS+7] and NIS, Neuropathy Symptom and Change, Polyneuropathy Disability), QoL (Norfolk QoL\u2010Diabetic Neuropathy), physical functioning (36\u2010Item Short\u2010Form Health Survey Physical Component Summary), nutritional status (modified body mass index), and serum transthyretin levels; these were compared with NEURO\u2010TTR historical placebo. Autonomic dysfunction (Composite Autonomic Symptom Score\u201031), disability (Rasch\u2010built Overall Disability Scale), and walking speed (10\u2010Meter Walk Test) were also assessed. Final assessments were carried out following 65\/66 or 81\/85\u2009weeks of treatment.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Mean mNIS+7 composite scores were maintained over 85\u2009weeks with eplontersen (changes from baseline of \u22124.5 [T1], \u22121.3 [T2], and \u22122.6 [T3] points). Other disease parameter scores were similarly maintained or improved with eplontersen. Patients receiving placebo experienced disease worsening across outcomes. T1 mean disease scores were typically better than in T2 and T3.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interpretation<\/jats:title>\n                    <jats:p>Consistent, sustained benefits of eplontersen were observed regardless of baseline ATTRv\u2010PN severity. These findings strengthen the importance of early treatment initiation for patients with ATTRv\u2010PN across the disease spectrum.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/jns.70167","type":"journal-article","created":{"date-parts":[[2026,9,5]],"date-time":"2026-09-05T02:46:08Z","timestamp":1788576368000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Impact of Baseline Polyneuropathy Severity on Eplontersen Efficacy in the\n                    <scp>NEURO<\/scp>\n                    \u2010\n                    <scp>TTRansform<\/scp>\n                    Clinical Trial"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1812-8331","authenticated-orcid":false,"suffix":"III","given":"Thomas H.","family":"Brannagan","sequence":"first","affiliation":[{"name":"Department of Neurology Columbia University, Vagelos College of Physicians and Surgeons  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8942-7662","authenticated-orcid":false,"given":"Markus","family":"Weiler","sequence":"additional","affiliation":[{"name":"Amyloidosis Center and Department of Neurology Heidelberg University Hospital  Heidelberg Germany"},{"name":"Faculty of Medicine Heidelberg University  Heidelberg Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4853-2236","authenticated-orcid":false,"given":"M\u00e1rcia Waddington","family":"Cruz","sequence":"additional","affiliation":[{"name":"CEPARM, Federal University of Rio de Janeiro  Rio de Janeiro Brazil"},{"name":"Amyloidosis Center Am\u00e9ricas\u2010Samaritano\u2010Vit\u00f3ria Hospital Complex  Rio de Janeiro Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5684-119X","authenticated-orcid":false,"given":"Sami","family":"Khella","sequence":"additional","affiliation":[{"name":"University of Pennsylvania School of Medicine  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1768-2373","authenticated-orcid":false,"given":"John L.","family":"Berk","sequence":"additional","affiliation":[{"name":"Boston University School of Medicine  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3853-5196","authenticated-orcid":false,"given":"Morie","family":"Gertz","sequence":"additional","affiliation":[{"name":"Department of Hematology Mayo Clinic  Rochester Minnesota USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6390-6526","authenticated-orcid":false,"given":"Ahmad","family":"Masri","sequence":"additional","affiliation":[{"name":"Oregon Health &amp; Science University  Portland Oregon USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5400-5008","authenticated-orcid":false,"given":"Mathew S.","family":"Maurer","sequence":"additional","affiliation":[{"name":"Columbia University Irving Medical Center  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1536-1277","authenticated-orcid":false,"given":"Jonas","family":"Wixner","sequence":"additional","affiliation":[{"name":"Amyloidosis Centre, Department of Public Health and Clinical Medicine Ume\u00e5 University Hospital  Ume\u00e5 Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-1604-2435","authenticated-orcid":false,"given":"Jonatan","family":"N\u00e5tman","sequence":"additional","affiliation":[{"name":"BioPharmaceuticals Business Unit AstraZeneca  Gothenburg Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-8726-3996","authenticated-orcid":false,"given":"T. Jesse","family":"Kwoh","sequence":"additional","affiliation":[{"name":"Clinical Development Ionis Pharmaceuticals, Inc  Carlsbad California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maksym","family":"Pola","sequence":"additional","affiliation":[{"name":"Late Cardiovascular, Renal, Metabolism BioPharmaceuticals R&amp;D, AstraZeneca  Gothenburg Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7468-700X","authenticated-orcid":false,"given":"Laura","family":"Obici","sequence":"additional","affiliation":[{"name":"Amyloidosis Research and Treatment Centre IRCCS Fondazione Policlinico San Matteo  Pavia Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,9,4]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.2147\/TCRM.S219979"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10741\u2010021\u201010080\u20102"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/s1474\u20104422(23)00334\u20104"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750\u20101172\u20108\u201031"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1136\/egastro\u20102025\u2010100198"},{"key":"e_1_2_10_7_1","unstructured":"US Food and Drug Administration \u201cWAINUA (Eplontersen). 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