{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T14:50:39Z","timestamp":1786978239576,"version":"build-2736575974"},"reference-count":32,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2026,4,6]],"date-time":"2026-04-06T00:00:00Z","timestamp":1775433600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,4,6]],"date-time":"2026-04-06T00:00:00Z","timestamp":1775433600000},"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":["Muscle and Nerve"],"published-print":{"date-parts":[[2026,6]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>Eplontersen is approved in multiple regions for adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv\u2010PN). This exploratory analysis was conducted to evaluate the treatment effect of eplontersen by sex in patients with ATTRv\u2010PN from the NEURO\u2010TTRansform trial (NCT04136184).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Participants in NEURO\u2010TTRansform who received \u2265\u20091 dose of eplontersen were included in this analysis; participants in the inotersen reference group who switched to eplontersen were excluded. Eplontersen was evaluated in the overall patient population and in a subgroup with cardiomyopathy. Change from baseline in the NEURO\u2010TTRansform primary endpoints of serum transthyretin (TTR) levels to Week 65, modified Neuropathy Impairment Score +7 (mNIS+7) composite score to Week 66, and Norfolk Quality of Life\u2010Diabetic Neuropathy (Norfolk QoL\u2010DN) total score to Week 66 were evaluated. A historical placebo group from the NEURO\u2010TTR trial (NCT01737398) was included as a control.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The eplontersen group comprised 100 (69.4%) male and 44 (30.6%) female patients. The placebo group comprised 41 (68.3%) male and 19 (31.7%) female patients. For both sexes, treatment with eplontersen decreased serum TTR levels by &gt;\u200980% (vs. reductions of 3%\u201313% with placebo), maintained mNIS+7 scores, and improved Norfolk QoL\u2010DN scores (vs. deterioration with placebo). The treatment effect of eplontersen was similar in male and female patients with cardiomyopathy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Eplontersen halted neuropathy impairment and improved quality of life versus placebo to a similar degree in both male and female patients with ATTRv\u2010PN, including those with cardiomyopathy. These findings support the use of eplontersen as an effective treatment for ATTRv\u2010PN regardless of sex.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70230","type":"journal-article","created":{"date-parts":[[2026,4,6]],"date-time":"2026-04-06T07:21:49Z","timestamp":1775460109000},"page":"1118-1127","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients"],"prefix":"10.1002","volume":"73","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4853-2236","authenticated-orcid":false,"given":"M\u00e1rcia","family":"Waddington Cruz","sequence":"first","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\/0000-0002-1768-2373","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\/0000-0001-9793-6590","authenticated-orcid":false,"given":"Ye\u015fim","family":"Parman","sequence":"additional","affiliation":[{"name":"\u0130stanbul \u00dcniversitesi \u2010 Istanbul T\u0131p Fak\u00fcltesi  Istanbul Turkey"}],"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\/0000-0001-5684-119X","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\/0000-0002-8942-7662","authenticated-orcid":false,"given":"Markus","family":"Weiler","sequence":"additional","affiliation":[{"name":"Amyloidosis Center and Department of Neurology Heidelberg University Hospital  Heidelberg Germany"}],"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"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-2130-3033","authenticated-orcid":false,"given":"Maksym","family":"Pola","sequence":"additional","affiliation":[{"name":"Late Cardiovascular, Renal, Metabolism BioPharmaceuticals R&amp;D  Gothenburg Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3420-7635","authenticated-orcid":false,"given":"Barry","family":"Reicher","sequence":"additional","affiliation":[{"name":"BioPharmaceuticals R&amp;D  Gaithersburg Maryland USA"}],"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  Gothenburg Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-5880-9646","authenticated-orcid":false,"given":"Noel R.","family":"Dasgupta","sequence":"additional","affiliation":[{"name":"Indiana University School of Medicine  Indianapolis Indiana 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  Ume\u00e5 Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,4,6]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-021-01960-9"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-023-02962-5"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10741-023-10339-w"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40119-022-00265-7"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/ejhf.2646"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-019-1000-1"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.126526"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1716793"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1080\/13506129.2022.2091985"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2300757"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.brainres.2008.06.095"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10571-008-9339-1"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11010-008-9841-2"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1038\/nri.2016.90"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41581-023-00787-w"},{"key":"e_1_2_12_17_1","unstructured":"US Food and Drug Administration \u201cEPLONTERSEN. Highlights of Prescribing Information \u201d2025https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2025\/217388Orig1s004lbl.pdf."},{"key":"e_1_2_12_18_1","unstructured":"Canadian Agency for Drugs and Technologies in Health \u201cEplontersen Approval \u201d2024https:\/\/www.cda\u2010amc.ca\/sites\/default\/files\/DRR\/2024\/SR0826REC\u2010Wainua_FINAL.pdf."},{"key":"e_1_2_12_19_1","unstructured":"European Medicines Agency \u201cWainzua (Eplontersen) an Overview of Wainzua and Why it is Authorised in the EU \u201d2025https:\/\/www.ema.europa.eu\/en\/documents\/overview\/wainzua\u2010epar\u2010medicine\u2010overview_en.pdf."},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2023.18688"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40120-021-00235-6"},{"key":"e_1_2_12_22_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40120-022-00414-z"},{"key":"e_1_2_12_23_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-024-12616-6"},{"key":"e_1_2_12_24_1","doi-asserted-by":"publisher","DOI":"10.1002\/ejhf.2824"},{"key":"e_1_2_12_25_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40120-014-0023-8"},{"key":"e_1_2_12_26_1","doi-asserted-by":"publisher","DOI":"10.1080\/13506129.2025.2577361"},{"key":"e_1_2_12_27_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-8-31"},{"key":"e_1_2_12_28_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jchf.2021.05.005"},{"key":"e_1_2_12_29_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-010-9673-x"},{"key":"e_1_2_12_30_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijerph18115951"},{"key":"e_1_2_12_31_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.archger.2019.05.009"},{"key":"e_1_2_12_32_1","doi-asserted-by":"publisher","DOI":"10.1210\/clinem\/dgab715"},{"key":"e_1_2_12_33_1","doi-asserted-by":"publisher","DOI":"10.14797\/mdcvj.1066"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.70230","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.70230","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.70230","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,4]],"date-time":"2026-05-04T14:36:11Z","timestamp":1777905371000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.70230"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4,6]]},"references-count":32,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2026,6]]}},"alternative-id":["10.1002\/mus.70230"],"URL":"https:\/\/doi.org\/10.1002\/mus.70230","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,4,6]]},"assertion":[{"value":"2025-08-07","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-03-23","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-04-06","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}