{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,28]],"date-time":"2026-03-28T06:18:19Z","timestamp":1774678699657,"version":"3.50.1"},"reference-count":37,"publisher":"Becaris Publishing Limited","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":[],"crossmark-restriction":false},"short-container-title":["J. Comp. Eff. Res."],"abstract":"<jats:p>\n            <jats:bold>Aim:<\/jats:bold>\n            To examine\u00a0benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression.\n            <jats:bold>Methods:<\/jats:bold>\n            Data from daily steroid users (placebo-treated) were pooled from four phase 2b\/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to 48\u00a0weeks in six-minute walk distance (6MWD), timed function tests\u00a0and North Star Ambulatory Assessment total score.\n            <jats:bold>Results:<\/jats:bold>\n            Among 231 patients receiving deflazacort (n\u00a0=\u00a0127) or prednisone (n\u00a0=\u00a0104), observed differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively older age (\u22658-years-old), greater disease progression (baseline timed stand from supine \u22655 s), or longer corticosteroid use (&gt;3\u00a0years).\n            <jats:bold>Conclusion:<\/jats:bold>\n            Daily deflazacort had greater benefits than daily prednisone particularly among older\/more progressed patients.\n          <\/jats:p>","DOI":"10.57264\/cer-2022-0190","type":"journal-article","created":{"date-parts":[[2023,2,21]],"date-time":"2023-02-21T00:52:43Z","timestamp":1676940763000},"source":"Crossref","is-referenced-by-count":6,"title":["Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status"],"prefix":"10.57264","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8779-3220","authenticated-orcid":false,"given":"Craig M","family":"McDonald","sequence":"first","affiliation":[{"name":"Departments of Physical Medicine &amp; Rehabilitation and Pediatrics, University of California \u2013 Davis, Davis, CA 95616, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8360-7558","authenticated-orcid":false,"given":"Jessica R","family":"Marden","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Perry B","family":"Shieh","sequence":"additional","affiliation":[{"name":"UCLA Health, Los Angeles, CA 90095, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brenda L","family":"Wong","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Massachusetts Memorial Medical Center Worcester, MA 01605, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Henry","family":"Lane","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Adina","family":"Zhang","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ha","family":"Nguyen","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5770-4266","authenticated-orcid":false,"given":"Molly","family":"Frean","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Panayiota","family":"Trifillis","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Karyn","family":"Koladicz","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"James","family":"Signorovitch","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA 02199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"36897","published-online":{"date-parts":[[2023,2,7]]},"reference":[{"issue":"3","key":"e_1_3_7_3_2","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1016\/S1474-4422(18)30024-3","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management","volume":"17","author":"Birnkrant DJ","year":"2018","unstructured":"Birnkrant DJ, Bushby K, Bann CM Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. 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