{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T00:58:51Z","timestamp":1791939531680,"version":"4.3.4"},"reference-count":12,"publisher":"Springer Science and Business Media LLC","issue":"7","license":[{"start":{"date-parts":[[2021,4,16]],"date-time":"2021-04-16T00:00:00Z","timestamp":1618531200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2021,4,16]],"date-time":"2021-04-16T00:00:00Z","timestamp":1618531200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Drugs"],"published-print":{"date-parts":[[2021,5]]},"DOI":"10.1007\/s40265-021-01512-2","type":"journal-article","created":{"date-parts":[[2021,4,16]],"date-time":"2021-04-16T13:10:27Z","timestamp":1618578627000},"page":"875-879","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":253,"title":["Casimersen: First Approval"],"prefix":"10.1007","volume":"81","author":[{"given":"Matt","family":"Shirley","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,4,16]]},"reference":[{"issue":"1","key":"1512_CR1","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1038\/s41572-021-00248-3","volume":"7","author":"D Duan","year":"2021","unstructured":"Duan D, Goemans N, Takeda S, et al. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021;7(1):13.","journal-title":"Nat Rev Dis Primers."},{"issue":"3","key":"1512_CR2","doi-asserted-by":"publisher","first-page":"251","DOI":"10.1016\/S1474-4422(18)30024-3","volume":"17","author":"DJ Birnkrant","year":"2018","unstructured":"Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251\u201367.","journal-title":"Lancet Neurol."},{"issue":"14","key":"1512_CR3","first-page":"1","volume":"20","author":"N Datta","year":"2020","unstructured":"Datta N, Ghosh PS. Update on muscular dystrophies with focus on novel treatments and biomarkers. Curr Neurol Neurosci Rep. 2020;20(14):1\u201312.","journal-title":"Curr Neurol Neurosci Rep."},{"key":"1512_CR4","volume-title":"Exon skipping and inclusion therapies. Methods in molecular biology","author":"M Rodrigues","year":"2018","unstructured":"Rodrigues M, Yokota T. An overview of recent advances and clinical applications of exon skipping and splice modulation for muscular dystrophy and various genetic diseases. In: Yokota T, Maruyama R, editors. Exon skipping and inclusion therapies. Methods in molecular biology, vol. 1828. New York: Humana Press; 2018."},{"issue":"7","key":"1512_CR5","doi-asserted-by":"publisher","first-page":"373","DOI":"10.1038\/s41582-019-0203-3","volume":"15","author":"IEC Verhaart","year":"2019","unstructured":"Verhaart IEC, Aartsma-Rus A. Therapeutic developments for Duchenne muscular dystrophy. Nat Rev Neurol. 2019;15(7):373\u201386.","journal-title":"Nat Rev Neurol."},{"key":"1512_CR6","unstructured":"US FDA. Amondys 45 (casimersen) injection, for intravenous use: US prescribing information. 2021. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2021\/213026lbl.pdf. Accessed 18 Mar 2021."},{"key":"1512_CR7","unstructured":"Sarepta Therapeutics Inc. Sarepta Therapeutics announces FDA approval of Amondys 45\u2122 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45 [media release]. 2021. https:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-therapeutics-announces-fda-approval-amondys-45tm. Accessed 18 Mar 2021."},{"key":"1512_CR8","unstructured":"US FDA. FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation [media release]. 2021. https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-approves-targeted-treatment-rare-duchenne-muscular-dystrophy-mutation-0. Accessed 18 Mar 2021."},{"key":"1512_CR9","unstructured":"Sarepta Therapeutics. Sarepta Therapeutics and University of Western Australia announce exclusive worldwide licensing agreement for exon-skipping program in Duchenne muscular dystrophy [media release]. 2013. https:\/\/investorrelations.sarepta.com\/static-files\/e20a099e-f99d-4237-8651-fa9c6d479090. Accessed 18 Mar 2021."},{"key":"1512_CR10","unstructured":"United States Securities and Exhange Commission. Sarepta Therapeutics, Inc.\u2014Form 10-K. 2018. https:\/\/www.sec.gov\/Archives\/edgar\/data\/873303\/000156459019005170\/srpt-10k_20181231.htm. Accessed 18 Mar 2021."},{"key":"1512_CR11","doi-asserted-by":"crossref","unstructured":"Wagner K, Kuntz N, Koenig E, et al. Casimersen treatment in patients with Duchenne muscular dystrophy: safety, tolerability, and pharmacokinetics over 144 weeks of treatment [poster P.288]. In: World Muscle Society Virtual Congress. 2020.","DOI":"10.1016\/S1096-7192(21)00237-7"},{"issue":"Suppl 1","key":"1512_CR12","doi-asserted-by":"publisher","first-page":"S130","DOI":"10.1016\/j.nmd.2020.08.285","volume":"30","author":"N Kuntz","year":"2020","unstructured":"Kuntz N, Wagner K, East L, et al. Casimersen treatment in eligible patients with Duchenne muscular dystrophy: safety, tolerability, and pharmacokinetics over 144 weeks of treatment [abstract no. P.288]. Neuromuscul Disord. 2020;30(Suppl 1):S130\u20131.","journal-title":"Neuromuscul Disord."}],"container-title":["Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-021-01512-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s40265-021-01512-2\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s40265-021-01512-2.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,5,10]],"date-time":"2021-05-10T10:05:33Z","timestamp":1620641133000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s40265-021-01512-2"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,4,16]]},"references-count":12,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2021,5]]}},"alternative-id":["1512"],"URL":"https:\/\/doi.org\/10.1007\/s40265-021-01512-2","relation":{},"ISSN":["0012-6667","1179-1950"],"issn-type":[{"value":"0012-6667","type":"print"},{"value":"1179-1950","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,4,16]]},"assertion":[{"value":"26 March 2021","order":1,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"16 April 2021","order":2,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The preparation of this review was not supported by any external funding.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Funding"}},{"value":"During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the author on the basis of scientific completeness and accuracy. M. Shirley is a salaried employee of Adis International Ltd\/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"Not applicable.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability"}}]}}