{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T14:27:40Z","timestamp":1790173660926,"version":"4.1.0"},"reference-count":31,"publisher":"SAGE Publications","issue":"8","license":[{"start":{"date-parts":[[2020,2,4]],"date-time":"2020-02-04T00:00:00Z","timestamp":1580774400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Ann Pharmacother"],"published-print":{"date-parts":[[2020,8]]},"abstract":"<jats:p>Objective: The objective of this article is to review the pharmacology, pharmacokinetics, efficacy, safety, dosage and administration, and formulary considerations of deflazacort. Data Sources: A search of MEDLINE and EMBASE (1946 to December 31, 2019) was conducted using the terms deflazacort and Duchenne muscular dystrophy (DMD). Results were limited to clinical trials, humans, and English. Additional sources and data were obtained from the references of included articles and prescribing information. Study Selection and Data Extraction: All articles published after July 2014 related to pharmacology, pharmacokinetics, efficacy, or safety of the therapy in human subjects were included. Data Synthesis: Deflazacort 0.9 mg\/kg\/d is a once-daily oral corticosteroid and is the first drug of its class to be Food and Drug Administration (FDA) approved for DMD. Studies with deflazacort show improved functional outcomes, delayed onset of cardiomyopathy, reduction in scoliosis surgery, and improved survival, but these improvements are supported by relatively weak evidence. Relevance to Patient Care and Clinical Practice: This review presents data from studies published after the most recent DMD 2016 treatment guidelines and offers prescribing considerations, including pharmacology, pharmacokinetics, adverse effects, formulary considerations, and areas of uncertainty. Conclusions: Deflazacort presents an additional, FDA-approved corticosteroid option for patients that offers improved quality of life for DMD patients. However, there is weak evidence to support these benefits; a full risk-benefit analysis considering adverse events, efficacy, cost, and previous trials of steroid therapy is necessary when selecting therapy. Further research will help clarify deflazacort\u2019s optimal dose, duration of treatment, and impact on quality of life.<\/jats:p>","DOI":"10.1177\/1060028019900500","type":"journal-article","created":{"date-parts":[[2020,2,5]],"date-time":"2020-02-05T02:22:07Z","timestamp":1580869327000},"page":"788-794","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":39,"title":["A Review of Deflazacort for Patients With Duchenne Muscular Dystrophy"],"prefix":"10.1177","volume":"54","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0026-7739","authenticated-orcid":false,"given":"Mary","family":"Bylo","sequence":"first","affiliation":[{"name":"MCPHS University, Worcester, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raechel","family":"Farewell","sequence":"additional","affiliation":[{"name":"MCPHS University, Worcester, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9275-7972","authenticated-orcid":false,"given":"Valerie A.","family":"Coppenrath","sequence":"additional","affiliation":[{"name":"MCPHS University, Worcester, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4142-3854","authenticated-orcid":false,"given":"Dinesh","family":"Yogaratnam","sequence":"additional","affiliation":[{"name":"MCPHS University, Worcester, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2020,2,4]]},"reference":[{"key":"bibr1-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0631-3"},{"key":"bibr2-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1002\/phar.1909"},{"key":"bibr3-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1111\/jpc.12868"},{"key":"bibr4-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002337"},{"key":"bibr5-1060028019900500","unstructured":"US Food and Drug Administration. FDA approves drug to treat Duchenne muscular dystrophy. https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-drug-treat-duchenne-muscular-dystrophy. Published February 9, 2017. Accessed October 16, 2018."},{"key":"bibr6-1060028019900500","unstructured":"Emflaza [package insert]. South Plainfield, NJ: PTC Ther-apeutics; 2019."},{"key":"bibr7-1060028019900500","doi-asserted-by":"publisher","DOI":"10.2165\/00003495-199550020-00008"},{"key":"bibr8-1060028019900500","first-page":"9","volume":"31","author":"Angelini C","year":"2012","journal-title":"Acta Myol"},{"key":"bibr9-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1023\/A:1016287104656"},{"key":"bibr10-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000001950"},{"key":"bibr11-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1177\/0883073814558120"},{"key":"bibr12-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2016.02.067"},{"key":"bibr13-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(17)32160-8"},{"key":"bibr14-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26191"},{"key":"bibr15-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1186\/s12872-017-0627-x"},{"key":"bibr16-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000003217"},{"key":"bibr17-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1016\/S0002-9149(02)03429-X"},{"key":"bibr18-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1016\/0960-8966(91)90099-E"},{"key":"bibr19-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1002\/1097-4598(200009)23:9<1344::AID-MUS4>3.0.CO;2-F"},{"key":"bibr20-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1007\/s00467-006-0403-0"},{"key":"bibr21-1060028019900500","doi-asserted-by":"publisher","DOI":"10.1021\/acs.jmedchem.5b00257"},{"key":"bibr22-1060028019900500","unstructured":"Lexi-Drugs: deflazacort. Lexicomp."},{"key":"bibr23-1060028019900500","unstructured":"Red Book: Emflaza. IBM Micromedex."},{"key":"bibr24-1060028019900500","unstructured":"Red Book: Prednisone. IBM Micromedex."},{"key":"bibr25-1060028019900500","unstructured":"Stewart J. Emflaza\u2019s new list price is $35K a year, but rises with patient\u2019s weight, PTC announces. https:\/\/musculardystrophynews.com\/2017\/05\/09\/emflaza-list-price-put-at-35000-by-ptc-on-weight-based-scale-reaction-swift\/. Published May 9, 2017. Accessed March 12, 2019."},{"key":"bibr26-1060028019900500","unstructured":"Institute for Clinical and Economic Review. Deflazacort, eteplirsen, and golodirsen for Duchenne muscular dystrophy: effectiveness and value. https:\/\/icer-review.org\/wp-content\/uploads\/2018\/12\/ICER_DMD_Draft_Evidence_Report_0522019.pdf. Published May 22, 2019. 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