{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T00:58:54Z","timestamp":1791939534692,"version":"4.3.4"},"reference-count":40,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,3,14]],"date-time":"2024-03-14T00:00:00Z","timestamp":1710374400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100014943","name":"Sarepta Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014943","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":[[2024,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>Eteplirsen, approved in the US for patients with Duchenne muscular dystrophy (DMD) with exon 51 skip\u2010amenable variants, is associated with attenuated ambulatory\/pulmonary decline versus DMD natural history (NH). We report overall survival in a US cohort receiving eteplirsen and contextualize these outcomes versus DMD NH.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>US patients with DMD receiving eteplirsen were followed through a patient support program, with data collected on ages at eteplirsen initiation and death\/end of follow\u2010up. Individual DMD NH data were extracted by digitizing Kaplan\u2013Meier (KM) curves from published systematic and targeted literature reviews. Overall survival age was analyzed using KM curves and contextualized with DMD NH survival curves; subanalyses considered age groups and duration of eteplirsen exposure. Overall survival time from treatment initiation was also evaluated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 579 eteplirsen\u2010treated patients were included. During a total follow\u2010up of 2119 person\u2010years, median survival age was 32.8\u2009years. DMD NH survival curves extracted from four publications (follow\u2010up for 1224 DMD NH controls) showed overall pooled median survival age of 27.4\u2009years. Eteplirsen\u2010treated patients had significantly longer survival from treatment initiation versus age\u2010matched controls (age\u2010adjusted hazard ratio [HR], 0.65; 95% confidence interval [CI], 0.44\u20130.98; <jats:italic>p<\/jats:italic>\u2009&lt;\u2009.05). Longer treatment exposure was associated with improved survival (HR, 0.15; 95% CI, 0.05\u20130.41; <jats:italic>p<\/jats:italic>\u2009&lt;\u2009.001). Comparisons using different DMD NH cohorts to address common risks of bias yielded consistent findings.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>Data suggest eteplirsen may prolong survival in patients with DMD across a wide age range. As more data become available, the impact of eteplirsen on survival will be further elucidated.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.28075","type":"journal-article","created":{"date-parts":[[2024,3,14]],"date-time":"2024-03-14T11:26:28Z","timestamp":1710415588000},"page":"60-70","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":29,"title":["Survival among patients receiving eteplirsen for up to 8\u2009years for the treatment of <scp>Duchenne<\/scp> muscular dystrophy and contextualization with natural history controls"],"prefix":"10.1002","volume":"70","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1102-0651","authenticated-orcid":false,"given":"Joel","family":"Iff","sequence":"first","affiliation":[{"name":"Sarepta Therapeutics, Inc.  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Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1536-8145","authenticated-orcid":false,"given":"Basil T.","family":"Darras","sequence":"additional","affiliation":[{"name":"Boston Children's Hospital and Harvard Medical School  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M.","family":"McDonald","sequence":"additional","affiliation":[{"name":"University of California Davis Health System  Sacramento California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugenio","family":"Mercuri","sequence":"additional","affiliation":[{"name":"Paediatric Neurology and Centro Clinico NeMO Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS  Rome Italy"},{"name":"Centro Clinico NeMO Fondazione Policlinico Universitario Agostino Gemelli IRCSS  Rome Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"Dubowitz Neuromuscular Centre UCL Institute of Child Health and Great Ormond Street Hospital for Children  London UK"},{"name":"Great Ormond Street Hospital  London UK"},{"name":"NIHR Great Ormond Street Hospital Biomedical Research Centre  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,3,14]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(18)30024\u20103"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010020\u201001430\u20108"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0218683"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318225ab2e"},{"key":"e_1_2_13_6_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0052512"},{"issue":"2","key":"e_1_2_13_7_1","first-page":"117","article-title":"Survival in Duchenne muscular dystrophy","volume":"31","author":"Rall S","year":"2012","journal-title":"Acta Myol"},{"key":"e_1_2_13_8_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41572\u2010021\u201000248\u20103"},{"key":"e_1_2_13_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(09)70272\u20108"},{"key":"e_1_2_13_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2010.09.006"},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD003725.pub4"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140\u20106736(17)32160\u20108"},{"key":"e_1_2_13_13_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00439\u2010019\u201002107\u20104"},{"key":"e_1_2_13_14_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00204\u2010021\u201003184\u2010z"},{"key":"e_1_2_13_15_1","unstructured":"Exondys 51 (eteplirsen) injection for intravenous use[Prescribing Information]. 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