{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T07:50:07Z","timestamp":1793346607530,"version":"4.5.0"},"reference-count":34,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2025,8,19]],"date-time":"2025-08-19T00:00:00Z","timestamp":1755561600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2025,8,19]],"date-time":"2025-08-19T00:00:00Z","timestamp":1755561600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"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":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2026,3]]},"abstract":"<jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>\n                      Duchenne muscular dystrophy (DMD) leads to dilated cardiomyopathy and heart failure during teenage years or young adulthood. Eteplirsen promotes dystrophin production through skipping of exon 51 of the\n                      <jats:italic toggle=\"yes\">DMD<\/jats:italic>\n                      gene.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>This analysis compared LVEF decline between eteplirsen-treated and control patients with exon 51 skip-amenable DMD.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Eteplirsen-treated patients from clinical trials were matched with control patients from natural history studies in a propensity score analysis. Risk of reaching LVEF thresholds (50%, 55%, and 60%) was evaluated using Cox proportional hazard models. Annual rate of LVEF decline was characterised using linear mixed effects models.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Among 141 eteplirsen-treated and 103 control patients available for matching, the analysis included 122 eteplirsen-treated patients matched with 122 control patients (64 unique control patients). No eteplirsen-treated and 27 controls (22.1%) reached LVEF &lt;50%; eteplirsen-treated patients had a lower risk of reaching &lt;55% and &lt;60% thresholds versus controls (hazard ratio\u2009=\u20090.22; 95% CI\u2009=\u20090.07\u20130.66;\n                      <jats:italic toggle=\"yes\">P<\/jats:italic>\n                      \u2009&lt;\u20090.01 and hazard ratio\u2009=\u20090.40; 95% CI\u2009=\u20090.22\u20130.76;\n                      <jats:italic toggle=\"yes\">P<\/jats:italic>\n                      \u2009&lt;\u20090.01, respectively). Annual rate of LVEF decline for eteplirsen-treated and controls was \u22120.66% (95% CI\u2009=\u2009\u20130.96 to \u22120.36,\n                      <jats:italic toggle=\"yes\">P<\/jats:italic>\n                      \u2009&lt;\u20090.01) and \u22121.38% (95% CI\u2009=\u2009\u20131.60 to \u22121.16;\n                      <jats:italic toggle=\"yes\">P<\/jats:italic>\n                      \u2009&lt;\u20090.01), respectively. Results were consistent with a sensitivity analysis matching each eteplirsen-treated patient once with a unique control patient and with several tests for potential bias.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>In this retrospective study, eteplirsen-treated patients were observed to have a significantly lower risk of reaching LVEF thresholds indicative of cardiac function decline and attenuation of LVEF decline compared with matched controls.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/22143602251366721","type":"journal-article","created":{"date-parts":[[2025,8,20]],"date-time":"2025-08-20T06:10:41Z","timestamp":1755670241000},"page":"179-189","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":8,"title":["Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy"],"prefix":"10.1177","volume":"13","author":[{"given":"Joel","family":"Iff","sequence":"first","affiliation":[{"name":"Sarepta Therapeutics, Inc"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Isabelle","family":"Desguerre","sequence":"additional","affiliation":[{"name":"APHP- Necker Hospital, Neuromuscular Center, FILNEMUS Network, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yunjuan","family":"Liu","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francois","family":"Sarkozy","sequence":"additional","affiliation":[{"name":"FSNB Health &amp; Care, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edward","family":"Tuttle","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"University College London, Great Ormond Street Institute of Child Health"},{"name":"National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M","family":"McDonald","sequence":"additional","affiliation":[{"name":"University of California Davis Health"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie-Christine","family":"Nougues","sequence":"additional","affiliation":[{"name":"H\u00f4pital Trousseau, APHP"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Helge","family":"Amthor","sequence":"additional","affiliation":[{"name":"H\u00f4pital Raymond Poincar\u00e9, APHP Universit\u00e9 Paris-Saclay"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yi","family":"Zhong","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karim","family":"Wahbi","sequence":"additional","affiliation":[{"name":"AP-HP, Cochin Hospital"},{"name":"Universit\u00e9 de Paris, Paris, France"},{"name":"Reference Center for Muscle Diseases Paris-Est, \rMyology Institute, AP-HP, Piti\u00e9-Salp\u00eatri\u00e8re Hospital, 75651, Paris 13, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study Investigators","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,8,19]]},"reference":[{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.2147\/DDDT.S97635"},{"key":"e_1_3_3_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ijcard.2023.131162"},{"key":"e_1_3_3_4_2","doi-asserted-by":"publisher","DOI":"10.1002\/humu.20918"},{"key":"e_1_3_3_5_2","doi-asserted-by":"publisher","DOI":"10.3390\/genes13071241"},{"key":"e_1_3_3_6_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-200548"},{"key":"e_1_3_3_7_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27662"},{"key":"e_1_3_3_8_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-210643"},{"key":"e_1_3_3_9_2","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2009.120"},{"key":"e_1_3_3_10_2","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2010.288"},{"key":"e_1_3_3_11_2","unstructured":"Wolford B. 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