{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,11]],"date-time":"2026-10-11T21:34:10Z","timestamp":1791754450023,"version":"4.3.4"},"reference-count":22,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2022,7,8]],"date-time":"2022-07-08T00:00:00Z","timestamp":1657238400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/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":[[2022,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>Pulmonary decline is a major issue in patients with Duchenne muscular dystrophy (DMD). Eteplirsen is a United States\u2013approved treatment for patients with DMD and exon 51 skip\u2010amenable mutations. Previous analyses have shown that eteplirsen is associated with a statistically significant attenuation of pulmonary decline. In this study we evaluate the effect of eteplirsen treatment from newly available data sources on pulmonary function over time in patients with DMD.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We used a post hoc pooled analysis to compare the percentage of predicted forced vital capacity (FVC%p) and projected time with pulmonary function milestones in patients with DMD and exon 51 skip\u2010amenable mutations receiving eteplirsen (Studies 204 and 301) or standard of care (SoC; Cooperative International Neuromuscular Research Group Duchenne Natural History Study). A mixed model for repeated\u2010measures framework was applied to evaluate the impact of eteplirsen.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>An average annual rate of FVC%p decline for eteplirsen\u2010treated patients was estimated to be 3.47%, a statistically significant attenuation from the 5.95% rate of decline estimated in SoC patients (<jats:italic>P<\/jats:italic>\u00a0=\u00a0.0001). Using linear extrapolations of the model\u2010estimated decline in FVC%p, the attenuation in FVC%p decline for eteplirsen\u2010treated patients corresponded to a delay of 5.72\u2009years in time to needing continuous ventilation, 3.31\u2009years in time to needing nighttime ventilation, and 2.11\u2009years in time to needing a cough assist device compared with SoC patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>The attenuation of FVC%p decline suggests that eteplirsen\u2010treated patients had statistically significant and clinically meaningful attenuations in pulmonary decline compared with SoC patients.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.27662","type":"journal-article","created":{"date-parts":[[2022,6,18]],"date-time":"2022-06-18T07:33:05Z","timestamp":1655537585000},"page":"262-269","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["Delays in pulmonary decline in eteplirsen\u2010treated patients with <scp>Duchenne<\/scp> muscular dystrophy"],"prefix":"10.1002","volume":"66","author":[{"given":"Joel","family":"Iff","sequence":"first","affiliation":[{"name":"Sarepta Therapeutics, Inc  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles","family":"Gerrits","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6246-2292","authenticated-orcid":false,"given":"Yi","family":"Zhong","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc  Menlo Park California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edward","family":"Tuttle","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc  Menlo Park California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erica","family":"Birk","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc  Menlo Park California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yeya","family":"Zheng","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc  Menlo Park California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xander","family":"Paul","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc  Menlo Park California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7511-6441","authenticated-orcid":false,"given":"Erik K.","family":"Henricson","sequence":"additional","affiliation":[{"name":"University of California Davis Medical Center  Sacramento California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M.","family":"McDonald","sequence":"additional","affiliation":[{"name":"University of California Davis Medical Center  Sacramento California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"CINRG\u2010DNHS Investigators","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,7,8]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-4939-7374-3_2"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-170272"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.200307-885ST"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pmr.2005.08.017"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30025-5"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1183\/20734735.0021711"},{"key":"e_1_2_12_8_1","volume-title":"Pulmonary Function Testing in Children: Techniques and Standards","author":"Polgar G"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1164\/ajrccm.159.1.9712108"},{"key":"e_1_2_12_10_1","first-page":"659","article-title":"Reference spirometric values using techniques and equipment that meet ATS recommendations","volume":"123","author":"Crapo RO","year":"1981","journal-title":"Am Rev Respir Dis"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2018.07.004"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.17925\/USN.2017.13.01.35"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.201703-0507WS"},{"key":"e_1_2_12_14_1","volume-title":"EXONDYS 51 (eteplirsen) Prescribing Information","year":"2022"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.24555"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-200548"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-180351"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejpn.2006.12.002"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23807"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.1080\/24745332.2017.1300463"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-170245"},{"key":"e_1_2_12_22_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejpn.2011.07.001"},{"key":"e_1_2_12_23_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27427"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27662","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.27662","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27662","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,22]],"date-time":"2023-08-22T05:29:50Z","timestamp":1692682190000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27662"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,7,8]]},"references-count":22,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2022,9]]}},"alternative-id":["10.1002\/mus.27662"],"URL":"https:\/\/doi.org\/10.1002\/mus.27662","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,7,8]]},"assertion":[{"value":"2021-09-10","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-06-11","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-07-08","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}