{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T19:09:40Z","timestamp":1790622580351,"version":"4.1.0"},"reference-count":31,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2019,11,7]],"date-time":"2019-11-07T00:00:00Z","timestamp":1573084800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013223","name":"PTC Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013223","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":[[2020,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>In this study we characterized disease progression over 48\u2009weeks among boys receiving deflazacort vs prednisone\/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Ambulatory boys with DMD receiving placebo in the phase 3 ataluren (N = 115) and tadalafil (N = 116) trials were included. The trials required at least 6\u2009months of prior corticosteroid use and stable baseline dosing. Associations between corticosteroid use and 48\u2010week changes in ambulatory function were estimated using mixed models. Adjusted differences between corticosteroid groups were pooled in a meta\u2010analysis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>In the meta\u2010analysis, deflazacort\u2010treated patients vs prednisone\/prednisolone\u2010treated patients experienced, on average, lower declines of 28.3 meters on 6\u2010minute walk distance (95% confidence interval [CI], 5.7, 50.9; 2.9\u2009seconds on rise from supine [95% CI, 0.9, 4.9\u2009seconds]; 2.3\u2009seconds on 4\u2010stair climb [95% CI, 0.5, 4.1\u2009seconds]; and 2.9 [95% CI, 0.1, 5.8] points on the North Star Ambulatory Assessment linearized score).<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>Deflazacort\u2010treated patients experienced significantly lower functional decline over 48\u2009weeks.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.26736","type":"journal-article","created":{"date-parts":[[2019,10,10]],"date-time":"2019-10-10T15:10:50Z","timestamp":1570720250000},"page":"26-35","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":69,"title":["Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta\u2010analysis of disease progression rates in recent multicenter clinical trials"],"prefix":"10.1002","volume":"61","author":[{"given":"Craig M.","family":"McDonald","sequence":"first","affiliation":[{"name":"Physical Medicine and Rehabilitation Pediatrics University of California Davis Health System  Sacramento California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gautam","family":"Sajeev","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc  Boston Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhiwen","family":"Yao","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc  Boston Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erin","family":"McDonnell","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc  Boston Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gary","family":"Elfring","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc  South Plainfield New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcio","family":"Souza","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc  South Plainfield New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stuart W.","family":"Peltz","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc  South Plainfield New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Basil T.","family":"Darras","sequence":"additional","affiliation":[{"name":"Department of Neurology Boston Children's Hospital  Boston Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Perry B.","family":"Shieh","sequence":"additional","affiliation":[{"name":"Neurology University of California at Los Angeles  Los Angeles California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David A.","family":"Cox","sequence":"additional","affiliation":[{"name":"Eli Lilly and Company  Indianapolis Indiana"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Landry","sequence":"additional","affiliation":[{"name":"Eli Lilly and Company  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James","family":"Signorovitch","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc  Boston Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"for the ACT DMD Study Group and the Tadalafil DMD Study Group","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2019,11,7]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.23528"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1093\/med\/9780199681488.001.0001"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002337"},{"key":"e_1_2_9_5_1","first-page":"CD003725","article-title":"Corticosteroids for the treatment of Duchenne muscular dystrophy","volume":"5","author":"Matthews E","year":"2016","journal-title":"Cochrane Database Syst Rev."},{"key":"e_1_2_9_6_1","first-page":"16","article-title":"The Canadian experience with long\u2010term deflazacort treatment in Duchenne muscular dystrophy","volume":"31","author":"McAdam 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metabolism of deflazacort in the rat, dog, monkey and man","volume":"171","author":"Assandri A","year":"1984","journal-title":"Adv Exp Med Biol."},{"key":"e_1_2_9_30_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23831"},{"key":"e_1_2_9_31_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24332"},{"key":"e_1_2_9_32_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.10.004"}],"container-title":["Muscle &amp; 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