{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,12]],"date-time":"2026-02-12T12:00:42Z","timestamp":1770897642992,"version":"3.50.1"},"reference-count":24,"publisher":"Becaris Publishing Limited","issue":"14","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."],"published-print":{"date-parts":[[2021,10]]},"abstract":"<jats:p>\n            <jats:bold>Aim:<\/jats:bold>\n            To describe reasons for switching from prednisone\/prednisolone to deflazacort and associated clinical outcomes among patients with Duchenne and Becker muscular dystrophy (DMD and BMD, respectively) in the\u00a0USA.\n            <jats:bold>Methods:<\/jats:bold>\n            A chart review of patients with DMD (n\u00a0=\u00a062) or BMD (n\u00a0=\u00a030) who switched from prednisone to deflazacort (02\/2017\u201312\/2018) collected demographic\/clinical characteristics, reasons for switching, outcomes and common adverse events.\n            <jats:bold>Results:<\/jats:bold>\n            The mean ages at switch were 20.1 (DMD) and 9.2 (BMD) years. The primary physician-reported reasons for switching were \u2018to slow disease progression\u2019 (DMD: 83%, BMD: 79%) and \u2018tolerability\u2019 (67 and 47%). Switching was \u2018very\u2019 or \u2018somewhat\u2019 effective at addressing the primary reasons in 90\u201395% of patients.\n            <jats:bold>Conclusion:<\/jats:bold>\n            Physician-reported outcomes were consistent with deflazacort addressing patients' primary reasons for switching.\n          <\/jats:p>","DOI":"10.2217\/cer-2021-0110","type":"journal-article","created":{"date-parts":[[2021,7,19]],"date-time":"2021-07-19T08:25:21Z","timestamp":1626683121000},"page":"1065-1078","source":"Crossref","is-referenced-by-count":4,"title":["Steroid switching in dystrophinopathy treatment: a US\u00a0chart review of patient characteristics and clinical outcomes"],"prefix":"10.57264","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8360-7558","authenticated-orcid":false,"given":"Jessica R","family":"Marden","sequence":"first","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claudio","family":"Santos","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc., South Plainfield, NJ\u00a007080, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Brian","family":"Pfister","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc., South Plainfield, NJ\u00a007080, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Richard","family":"Able","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc., South Plainfield, NJ\u00a007080, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Henry","family":"Lane","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael","family":"Somma","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jing","family":"Zhao","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"James","family":"Signorovitch","sequence":"additional","affiliation":[{"name":"Analysis Group, Inc., Boston, MA\u00a002199, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julie","family":"Parsons","sequence":"additional","affiliation":[{"name":"Children's Hospital Colorado, Aurora, CO\u00a080045, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Susan","family":"Apkon","sequence":"additional","affiliation":[{"name":"Children's Hospital Colorado, Aurora, CO\u00a080045, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"36897","reference":[{"issue":"6","key":"e_1_3_7_3_2","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1016\/0092-8674(87)90579-4","article-title":"Dystrophin: the protein product of the Duchenne muscular dystrophy locus","volume":"51","author":"Hoffman EP","year":"1987","unstructured":"Hoffman EP, Brown RH Jr, Kunkel LM. 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