{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,21]],"date-time":"2026-09-21T23:15:02Z","timestamp":1790032502235,"version":"4.0.1"},"reference-count":32,"publisher":"SAGE Publications","issue":"2","license":[{"start":{"date-parts":[[2019,2,23]],"date-time":"2019-02-23T00:00:00Z","timestamp":1550880000000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2019,2,23]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Duchenne muscular dystrophy (DMD) patients experience skeletal muscle degeneration, including respiratory muscles. Respiratory decline in glucocorticoid-treated DMD patients, measured by percent predicted forced vital capacity (FVC% p), is typically 5% annually in patients aged 10 to 18 years.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>Evaluate the effects of eteplirsen on FVC% p annual change in 3 trials versus matched Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG DNHS) controls.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Eteplirsen studies 201\/202 evaluated eligible ambulatory DMD patients for at least 4 years, study 204 evaluated primarily non-ambulatory DMD patients for 2 years, and ongoing study 301 is evaluating ambulatory DMD patients for 2 years (interim analysis is included). Eteplirsen-treated patients ( n\u200a=\u200a74) were amenable to exon 51 skipping and were receiving glucocorticoids. Three CINRG DNHS cohorts included: glucocorticoid-treated patients amenable to exon 51 skipping (Exon 51 CINRG DNHS; n\u200a=\u200a20), all glucocorticoid-treated CINRG patients (All CINRG DNHS; n\u200a=\u200a172), and all glucocorticoid-treated genotyped CINRG DNHS patients (Genotyped CINRG DNHS; n\u200a=\u200a148). FVC% p assessments between ages 10 and\u200a&lt;18 years were included for all patients; mixed-model analyses characterized FVC% p annual change.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>FVC% p annual change was greater for CINRG DNHS Exon 51 controls (\u2013 6.00) versus patients in studies 201\/202, study 204, and study 301 (\u2013 2.19, P\u200a&lt;\u200a0.001; \u2013 3.66, P 0.004; and \u2013 3.79, P 0.017, respectively). FVC% p annual change in all eteplirsen studies suggested treatment benefit compared with the Genotyped CINRG DNHS (\u2013 5.67) and All CINRG DNHS (\u2013 5.56) cohorts ( P\u200a&lt;\u200a0.05, all comparisons).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions:<\/jats:title>\n                    <jats:p>Significant, clinically meaningful attenuation of FVC%p decline was observed in eteplirsen-treated patients versus CINRG DNHS controls.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3233\/jnd-180351","type":"journal-article","created":{"date-parts":[[2019,3,8]],"date-time":"2019-03-08T11:44:21Z","timestamp":1552045461000},"page":"213-225","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":91,"title":["Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy"],"prefix":"10.1177","volume":"6","author":[{"given":"Navid","family":"Khan","sequence":"first","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Helen","family":"Eliopoulos","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lixin","family":"Han","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"T. Bernard","family":"Kinane","sequence":"additional","affiliation":[{"name":"Massachusetts General Hospital for Children, Harvard Medical School, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda P.","family":"Lowes","sequence":"additional","affiliation":[{"name":"Nationwide Children\u2019s Hospital, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerry R.","family":"Mendell","sequence":"additional","affiliation":[{"name":"Nationwide Children\u2019s Hospital, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heather","family":"Gordish-Dressman","sequence":"additional","affiliation":[{"name":"Center for Genetic Medicine, Children\u2019s National Health System and the George Washington University School of Medicine and Health Sciences, Washington DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erik K.","family":"Henricson","sequence":"additional","affiliation":[{"name":"University of California Davis Health, Sacramento, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M.","family":"McDonald","sequence":"additional","affiliation":[{"name":"University of California Davis Health, Sacramento, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"on behalf of the Eteplirsen Investigators and the CINRG DNHS Investigators","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2019,3,7]]},"reference":[{"key":"ref001","doi-asserted-by":"publisher","DOI":"10.1097\/00002060-199509001-00003"},{"key":"ref002","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"ref003","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2013.04.005"},{"key":"ref004","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM198805263182104"},{"key":"ref005","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002147"},{"key":"ref006","doi-asserted-by":"publisher","DOI":"10.1085\/jgp.201010436"},{"issue":"1","key":"ref007","first-page":"7","volume":"13","author":"Mayer O","year":"2017","journal-title":"US Neurology"},{"key":"ref008","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)32160-8"},{"key":"ref009","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70272-8"},{"key":"ref010","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2018.07.004"},{"key":"ref011","doi-asserted-by":"publisher","DOI":"10.1016\/S0003-9993(97)90001-0"},{"key":"ref012","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.22847"},{"key":"ref013","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.23172"},{"key":"ref014","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.1988.tb04751.x"},{"key":"ref015","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(18)30025-5"},{"key":"ref016","doi-asserted-by":"publisher","DOI":"10.1002\/humu.20918"},{"key":"ref017","doi-asserted-by":"publisher","DOI":"10.1002\/humu.22758"},{"key":"ref018","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002891"},{"issue":"2","key":"ref019","first-page":"149","volume":"87","author":"Ricotti V","year":"2016","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"ref020","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23982"},{"key":"ref021","doi-asserted-by":"publisher","DOI":"10.3233\/JND-170272"},{"key":"ref022","doi-asserted-by":"publisher","DOI":"10.1002\/mus.880040304"},{"key":"ref023","doi-asserted-by":"publisher","DOI":"10.1111\/j.1469-8749.2004.tb00508.x"},{"key":"ref024","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23807"},{"key":"ref025","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23808"},{"key":"ref026","unstructured":"Duchenne Natural History. 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