{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T14:27:13Z","timestamp":1790173633744,"version":"4.1.0"},"reference-count":34,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2018,7,11]],"date-time":"2018-07-11T00:00:00Z","timestamp":1531267200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","award":["UL1TR001070"],"award-info":[{"award-number":["UL1TR001070"]}],"id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"published-print":{"date-parts":[[2018,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>This double\u2010blind, randomized, placebo\u2010controlled Phase 2 study (<jats:styled-content style=\"fixed-case\">NCT<\/jats:styled-content>01462292) assessed the 24\u2010week efficacy, safety, tolerability, and pharmacokinetics of two different subcutaneous drisapersen doses, and the 24\u2010week off\u2010dose persistent effect, in ambulant Duchenne muscular dystrophy (<jats:styled-content style=\"fixed-case\">DMD<\/jats:styled-content>) patients.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Male <jats:styled-content style=\"fixed-case\">DMD<\/jats:styled-content> patients (\u22655 years; time to rise from floor \u226415 s) were randomized to drisapersen 3 mg\/kg\/week, 6 mg\/kg\/week or placebo. The primary efficacy endpoint was change from baseline in 6\u2010minute walking distance (6<jats:styled-content style=\"fixed-case\">MWD<\/jats:styled-content>) at week 24. Secondary endpoints included changes in timed function tests, muscle strength, and pulmonary function tests.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Fifty\u2010one patients were randomized to placebo (<jats:italic>N<\/jats:italic> = 16), drisapersen 3 mg\/kg\/week (<jats:italic>N<\/jats:italic> = 17) or 6 mg\/kg\/week (<jats:italic>N<\/jats:italic> = 18). All but 2 patients had baseline rise from floor time &lt;7 s. This study was exploratory and not prospectively powered; however, a difference in mean 6<jats:styled-content style=\"fixed-case\">MWD<\/jats:styled-content> versus placebo in favor of drisapersen 6 mg\/kg\/week was observed at week 24 (27.1 m; <jats:italic>P<\/jats:italic> = 0.069) and maintained 24 weeks off\u2010treatment (27.9 m; <jats:italic>P<\/jats:italic> = 0.177). The 3 mg\/kg\/week group showed no statistically significant difference in mean 6<jats:styled-content style=\"fixed-case\">MWD<\/jats:styled-content> versus placebo. For some secondary endpoints, a more positive response in favor of drisapersen 6 mg\/kg\/week compared to placebo was shown. Drisapersen had a long half\u2010life with steady state reached after approximately 36 weeks. Most common adverse events in both drisapersen groups were related to injection site reactions and subclinical proteinuria.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>Drisapersen 6 mg\/kg\/week for 24 weeks resulted in a treatment benefit in 6<jats:styled-content style=\"fixed-case\">MWD<\/jats:styled-content>, largely maintained 24 weeks off\u2010treatment. 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