{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T07:18:35Z","timestamp":1791443915074,"version":"4.3.3"},"reference-count":47,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2016,1,8]],"date-time":"2016-01-08T00:00:00Z","timestamp":1452211200000},"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":"crossref","id":[{"id":"10.13039\/100014943","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Annals of Neurology"],"published-print":{"date-parts":[[2016,2]]},"abstract":"<jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>\n                      To continue evaluation of the long\u2010term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip\n                      <jats:italic>DMD<\/jats:italic>\n                      exon 51 in patients with Duchenne muscular dystrophy (DMD). Three\u2010year progression of eteplirsen\u2010treated patients was compared to matched historical controls (HC).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Ambulatory DMD patients who were \u22657 years old and amenable to exon 51 skipping were randomized to eteplirsen (30\/50mg\/kg) or placebo for 24 weeks. Thereafter, all received eteplirsen on an open\u2010label basis. The primary functional assessment in this study was the 6\u2010Minute Walk Test (6MWT). Respiratory muscle function was assessed by pulmonary function testing (PFT). Longitudinal natural history data were used for comparative analysis of 6MWT performance at baseline and months 12, 24, and 36. Patients were matched to the eteplirsen group based on age, corticosteroid use, and genotype.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      At 36 months, eteplirsen\u2010treated patients (n = 12) demonstrated a statistically significant advantage of 151m (\n                      <jats:italic>p<\/jats:italic>\n                      &lt; 0.01) on 6MWT and experienced a lower incidence of loss of ambulation in comparison to matched HC (n = 13) amenable to exon 51 skipping. PFT results remained relatively stable in eteplirsen\u2010treated patients. Eteplirsen was well tolerated. Analysis of HC confirmed the previously observed change in disease trajectory at age 7 years, and more severe progression was observed in patients with mutations amenable to exon skipping than in those not amenable. The subset of patients amenable to exon 51 skipping showed a more severe disease course than those amenable to any exon skipping.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interpretation<\/jats:title>\n                    <jats:p>Over 3 years of follow\u2010up, eteplirsen\u2010treated patients showed a slower rate of decline in ambulation assessed by 6MWT compared to untreated matched HC. Ann Neurol 2016;79:257\u2013271<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/ana.24555","type":"journal-article","created":{"date-parts":[[2015,11,17]],"date-time":"2015-11-17T02:32:58Z","timestamp":1447727578000},"page":"257-271","source":"Crossref","is-referenced-by-count":500,"title":["Longitudinal effect of eteplirsen versus historical control on ambulation in\n                    <scp>D<\/scp>\n                    uchenne muscular dystrophy"],"prefix":"10.1002","volume":"79","author":[{"given":"Jerry R.","family":"Mendell","sequence":"first","affiliation":[{"name":"Nationwide Children's Hospital Columbus OH"},{"name":"Department of Pediatrics and Neurology Ohio State University Columbus OH"},{"name":"Paul D. 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