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Exon skipping in these patients has the potential to be highly therapeutic through restoration of full-length dystrophin expression. We conducted a 48-week open label study of casimersen and golodirsen in 3 subjects with an exon 45 or 53 duplication. Two subjects (aged 18 and 23 years) were non-ambulatory at baseline. Upper limb, pulmonary, and cardiac function appeared stable in the 2 subjects in whom they could be evaluated. Dystrophin expression increased from 0.94\u200a% \u00b10.59% (mean\u00b1SD) of normal to 5.1% \u00b12.9% by western blot. Percent dystrophin positive fibers also rose from 14% \u00b117% at baseline to 50% \u00b142% . Our results provide initial evidence that the use of exon-skipping drugs may increase dystrophin levels in patients with single-exon duplications.<\/jats:p>","DOI":"10.3233\/jnd-230107","type":"journal-article","created":{"date-parts":[[2024,3,8]],"date-time":"2024-03-08T10:20:29Z","timestamp":1709893229000},"page":"679-685","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":8,"title":["Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study"],"prefix":"10.1177","volume":"11","author":[{"given":"Stefan","family":"Nicolau","sequence":"first","affiliation":[{"name":"Center for Gene Therapy, Nationwide Children\u2019s Hospital, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jyoti","family":"Malhotra","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics Inc., Cambridge, MA, 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