{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T18:26:04Z","timestamp":1787250364773,"version":"build-2736575974"},"reference-count":41,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2026,3,4]],"date-time":"2026-03-04T00:00:00Z","timestamp":1772582400000},"content-version":"vor","delay-in-days":3,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2026,3,1]],"date-time":"2026-03-01T00:00:00Z","timestamp":1772323200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Health Science Reports"],"published-print":{"date-parts":[[2026,3]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Hereditary transthyretin\u2010mediated amyloidosis (hATTR) is a disorder that affects several body systems and can result in life\u2010threatening conditions like cardiomyopathy and polyneuropathy. For treatment of hATTR in the new medical realm, there is a breakthrough discovery of a medicine called Eplontersen, which targets the underlying genetic mutation causing the condition. This review aims to summarize Eplontersen's mechanism of action, efficacy, safety profile, and ongoing clinical trials.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A systematic review of the literature was conducted following PRISMA guidelines. Databases searched included PubMed, Cochrane, and Science Direct using terms related to Eplontersen and hereditary amyloidosis. Studies were selected based on defined inclusion and exclusion criteria.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Eplontersen was approved by the FDA after the NEURO\u2010TTRansform phase 3 trial showed substantial safety and efficacy. The drug demonstrated a significant reduction in serum TTR levels and improvements in neurological function and quality of life in patients with hATTR. The review also explored contraindications, dosing, safety monitoring, and patient support programs.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Although data gaps remain, Eplontersen represents a promising advancement in the treatment of hATTR\u2010induced polyneuropathy. Future research is needed on long\u2010term safety, early diagnosis, personalized treatments, and combination therapies. This drug emphasizes the importance of continued research for optimal patient care and outcomes.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/hsr2.72004","type":"journal-article","created":{"date-parts":[[2026,3,8]],"date-time":"2026-03-08T14:51:39Z","timestamp":1772981499000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Therapeutic Efficacy and Safety Profile of Eplontersen in Hereditary Transthyretin\u2010Mediated Amyloidosis: A Systematic Review"],"prefix":"10.1002","volume":"9","author":[{"given":"Zeel Vishnubhai","family":"Patel","sequence":"first","affiliation":[{"name":"Internal Medicine Medical College Baroda  Gujarat India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Verkha","family":"Kumari","sequence":"additional","affiliation":[{"name":"Liaquat National Hospital and Medical College Karachi Pakistan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"family":"Paras","sequence":"additional","affiliation":[{"name":"Internal Medicine Government Medical College Patiala  Punjab India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3637-3768","authenticated-orcid":false,"given":"Bethineedi Lakshmi","family":"Deepak","sequence":"additional","affiliation":[{"name":"Andhra Medical College Visakhapatnam Andhra Pradesh India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7894-1829","authenticated-orcid":false,"given":"Priyadarshi","family":"Prajjwal","sequence":"additional","affiliation":[{"name":"Internal Medicine Bharati Vidyapeeth University Medical College Pune Maharashtra India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kolanu Nikhil","family":"Deep","sequence":"additional","affiliation":[{"name":"Internal Medicine China Medical University Shenyang China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rakhi","family":"Bai","sequence":"additional","affiliation":[{"name":"Liaquat National Hospital and Medical College Karachi Pakistan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-4792-716X","authenticated-orcid":false,"given":"Serene Sara","family":"John","sequence":"additional","affiliation":[{"name":"Internal Medicine Bharati Vidyapeeth Medical College Sangli India"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5021-263X","authenticated-orcid":false,"given":"Omniat Amir","family":"Hussin","sequence":"additional","affiliation":[{"name":"Internal Medicine Almanhal University Khartoum Sudan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,3,4]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41582-019-0210-4"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1080\/13506129.2022.2091985"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1056\/nejmoa1716793"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/s1474-4422(20)30368-9"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2023.18688"},{"key":"e_1_2_13_7_1","unstructured":"CARDIO\u2010TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION\u2010682884 IONIS\u2010TTR\u2010LRx and AKCEA\u2010TTR\u2010LRx) in Participants With Transthyretin\u2010Mediated Amyloid Cardiomyopathy (ATTR CM). (n.d.). Retrieved April 16 2024 from Clinicaltrials.gov website https:\/\/classic.clinicaltrials.gov\/ct2\/show\/NCT04136171."},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40120-022-00414-z"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.15468"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1161\/jaha.123.030512"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cardfail.2023.11.016"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1002\/ehf2.13154"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2014-308724"},{"key":"e_1_2_13_14_1","doi-asserted-by":"publisher","DOI":"10.1093\/eurheartjsupp\/suac104"},{"key":"e_1_2_13_15_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40259-023-00577-7"},{"key":"e_1_2_13_16_1","doi-asserted-by":"publisher","DOI":"10.3390\/ph17020243"},{"key":"e_1_2_13_17_1","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2018.0753"},{"key":"e_1_2_13_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/s0140-6736(16)31009-1"},{"key":"e_1_2_13_19_1","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gku531"},{"key":"e_1_2_13_20_1","doi-asserted-by":"publisher","DOI":"10.1080\/17425255.2019.1621838"},{"key":"e_1_2_13_21_1","doi-asserted-by":"publisher","DOI":"10.1056\/nejmoa1701329"},{"key":"e_1_2_13_22_1","doi-asserted-by":"publisher","DOI":"10.1093\/eurheartj\/ehz209"},{"key":"e_1_2_13_23_1","doi-asserted-by":"publisher","DOI":"10.1038\/mtna.2016.26"},{"key":"e_1_2_13_24_1","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2016.0623"},{"key":"e_1_2_13_25_1","doi-asserted-by":"publisher","DOI":"10.1056\/nejmoa1905239"},{"key":"e_1_2_13_26_1","doi-asserted-by":"publisher","DOI":"10.1111\/ene.14285"},{"key":"e_1_2_13_27_1","unstructured":"\u201cTegsedi.com \u201dTEGSEDI\u2010Inotersen Injection Solution accessed April 16 2024 https:\/\/tegsedi.com\/pdf\/prescribing-information.pdf."},{"key":"e_1_2_13_28_1","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2019.0829"},{"key":"e_1_2_13_29_1","doi-asserted-by":"publisher","DOI":"10.1517\/17425255.2013.737320"},{"key":"e_1_2_13_30_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtn.2017.08.012"},{"key":"e_1_2_13_31_1","volume-title":"Hereditary Transthyretin Amyloidosis","author":"Sekijima Y.","year":"2021"},{"key":"e_1_2_13_32_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-8-31"},{"key":"e_1_2_13_33_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40120-021-00235-6"},{"key":"e_1_2_13_34_1","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2022.0044"},{"key":"e_1_2_13_35_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2013.283815"},{"key":"e_1_2_13_36_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1716153"},{"key":"e_1_2_13_37_1","doi-asserted-by":"publisher","DOI":"10.1111\/jns.12519"},{"key":"e_1_2_13_38_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2019-129269"},{"key":"e_1_2_13_39_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.circv.2024.11.009"},{"key":"e_1_2_13_40_1","doi-asserted-by":"publisher","DOI":"10.4103\/heartviews.heartviews_100_24"},{"key":"e_1_2_13_41_1","doi-asserted-by":"publisher","DOI":"10.47363\/JCRRR\/2024(5)192"},{"key":"e_1_2_13_42_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.hrtlng.2021.03.065"}],"container-title":["Health Science Reports"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/hsr2.72004","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/hsr2.72004","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/hsr2.72004","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T17:53:59Z","timestamp":1787248439000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/hsr2.72004"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,3]]},"references-count":41,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,3]]}},"alternative-id":["10.1002\/hsr2.72004"],"URL":"https:\/\/doi.org\/10.1002\/hsr2.72004","archive":["Portico"],"relation":{},"ISSN":["2398-8835","2398-8835"],"issn-type":[{"value":"2398-8835","type":"print"},{"value":"2398-8835","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,3]]},"assertion":[{"value":"2025-04-16","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-02-23","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-03-04","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e72004"}}