{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,20]],"date-time":"2026-01-20T13:39:39Z","timestamp":1768916379911,"version":"3.49.0"},"reference-count":46,"publisher":"Informa UK Limited","license":[{"start":{"date-parts":[[2021,12,1]],"date-time":"2021-12-01T00:00:00Z","timestamp":1638316800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/3.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["CPAA"],"DOI":"10.2147\/cpaa.s288842","type":"journal-article","created":{"date-parts":[[2021,12,15]],"date-time":"2021-12-15T11:40:46Z","timestamp":1639568446000},"page":"235-242","source":"Crossref","is-referenced-by-count":9,"title":["Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience"],"prefix":"10.2147","volume":"Volume 13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6415-6862","authenticated-orcid":true,"given":"Rohini Roy","family":"Roshmi","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6672-6742","authenticated-orcid":true,"given":"Toshifumi","family":"Yokota","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"301","published-online":{"date-parts":[[2021,12]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","first-page":"304","DOI":"10.1002\/ana.23528","volume":"71","author":"Mendell","year":"2012","journal-title":"Ann Neurol"},{"key":"ref2","doi-asserted-by":"publisher","first-page":"919","DOI":"10.1016\/0092-8674(87)90579-4","volume":"51","author":"Hoffman","year":"1987","journal-title":"Cell"},{"key":"ref3","doi-asserted-by":"publisher","first-page":"466","DOI":"10.1038\/333466a0","volume":"333","author":"Zubrzycka-Gaarn","year":"1988","journal-title":"Nature"},{"key":"ref4","doi-asserted-by":"publisher","first-page":"986","DOI":"10.1136\/adc.2007.118141","volume":"93","author":"Manzur","year":"2008","journal-title":"Arch Dis Child"},{"key":"ref5","doi-asserted-by":"publisher","first-page":"1795","DOI":"10.2147\/NDT.S93873","volume":"12","author":"Mah","year":"2016","journal-title":"Neuropsychiatr Dis Treat"},{"key":"ref6","doi-asserted-by":"publisher","first-page":"509","DOI":"10.1016\/0092-8674(87)90504-6","volume":"50","author":"Koenig","year":"1987","journal-title":"Cell"},{"key":"ref7","doi-asserted-by":"publisher","first-page":"774","DOI":"10.1038\/nrg1180","volume":"4","author":"van Deutekom","year":"2003","journal-title":"Nat Rev Genet"},{"key":"ref8","doi-asserted-by":"publisher","first-page":"e0135189","DOI":"10.1371\/journal.pone.0135189","volume":"10","author":"Juan-Mateu","year":"2015","journal-title":"PLoS One"},{"key":"ref9","doi-asserted-by":"publisher","first-page":"599","DOI":"10.1093\/hmg\/1.8.599","volume":"1","author":"Oudet","year":"1992","journal-title":"Hum Mol Genet"},{"key":"ref10","doi-asserted-by":"publisher","first-page":"731","DOI":"10.1016\/S1474-4422(03)00585-4","volume":"2","author":"Muntoni","year":"2003","journal-title":"Lancet Neurol"},{"key":"ref11","doi-asserted-by":"publisher","first-page":"579","DOI":"10.1590\/0004-282x20190088","volume":"77","author":"Werneck","year":"2019","journal-title":"Arq Neuropsiquiatr"},{"key":"ref12","doi-asserted-by":"publisher","first-page":"1046","DOI":"10.1016\/j.nmd.2012.07.009","volume":"22","author":"Rutter","year":"2012","journal-title":"Neuromuscul Disord"},{"key":"ref13","doi-asserted-by":"publisher","first-page":"1409","DOI":"10.1016\/S0140-6736(74)90071-3","volume":"304","author":"Drachman","year":"1974","journal-title":"Lancet"},{"key":"ref14","doi-asserted-by":"publisher","first-page":"101","DOI":"10.1136\/archdischild-2015-308654","volume":"101","author":"Wood","year":"2016","journal-title":"Arch Dis Child"},{"key":"ref15","doi-asserted-by":"publisher","first-page":"R163","DOI":"10.1093\/hmg\/ddy171","volume":"27","author":"Echevarr\u00eda","year":"2018","journal-title":"Hum Mol Genet"},{"key":"ref16","doi-asserted-by":"publisher","first-page":"167","DOI":"10.1080\/13543784.2021.1868434","volume":"30","author":"Sheikh","year":"2021","journal-title":"Expert Opin Investig Drugs"},{"key":"ref17","doi-asserted-by":"publisher","first-page":"373","DOI":"10.1038\/s41582-019-0203-3","volume":"15","author":"Verhaart","year":"2019","journal-title":"Nat Rev Neurol"},{"key":"ref18","doi-asserted-by":"publisher","first-page":"67","DOI":"10.1089\/nat.2020.0845","volume":"30","author":"Aartsma-Rus","year":"2020","journal-title":"Nucleic Acid Ther"},{"key":"ref19","unstructured":"Casimersen (Amondys 45) for Duchenne muscular dystrophy. Med Lett Drugs Ther. 2021;63(1627):e104\u2013e105. Available from: http:\/\/www.ncbi.nlm.nih.gov\/pubmed\/34181634. Accessed November 15, 2021."},{"key":"ref20","doi-asserted-by":"publisher","first-page":"875","DOI":"10.1007\/s40265-021-01512-2","volume":"81","author":"Shirley","year":"2021","journal-title":"Drugs"},{"key":"ref21","doi-asserted-by":"publisher","first-page":"627","DOI":"10.1358\/dot.2019.55.10.3045038","volume":"55","author":"Roshmi","year":"2019","journal-title":"Drugs Today"},{"key":"ref22","doi-asserted-by":"publisher","first-page":"1027","DOI":"10.1007\/s40265-020-01339-3","volume":"80","author":"Dhillon","year":"2020","journal-title":"Drugs"},{"key":"ref23","doi-asserted-by":"publisher","first-page":"533","DOI":"10.2147\/DDDT.S97635","volume":"11","author":"Lim","year":"2017","journal-title":"Drug Des Devel Ther"},{"key":"ref24","doi-asserted-by":"publisher","DOI":"10.3389\/fmicb.2018.00750","volume":"9","author":"Nan","year":"2018","journal-title":"Front Microbiol"},{"key":"ref25","doi-asserted-by":"publisher","volume-title":"Comprehensive Physiology","year":"2015","DOI":"10.1002\/cphy.c140048"},{"key":"ref26","doi-asserted-by":"publisher","DOI":"10.17305\/bjbms.2015.636","volume":"15","author":"Le Rumeur","year":"2015","journal-title":"Bosn J Basic Med Sci"},{"key":"ref27","doi-asserted-by":"publisher","first-page":"667","DOI":"10.1002\/ana.21627","volume":"65","author":"Yokota","year":"2009","journal-title":"Ann Neurol"},{"key":"ref28","doi-asserted-by":"publisher","first-page":"442","DOI":"10.1016\/j.omtn.2018.09.017","volume":"13","author":"Watanabe","year":"2018","journal-title":"Mol Ther Nucleic Acids"},{"key":"ref29","doi-asserted-by":"publisher","first-page":"329","DOI":"10.1007\/s40265-020-01267-2","volume":"80","author":"Heo","year":"2020","journal-title":"Drugs"},{"key":"ref30","doi-asserted-by":"crossref","unstructured":"Viltolarsen. Am J Health Pharm. 2020;77(24):2032\u20132033. doi:10.1093\/ajhp\/zxaa324","DOI":"10.1093\/ajhp\/zxaa324"},{"key":"ref31","doi-asserted-by":"publisher","first-page":"2393","DOI":"10.1002\/acn3.51235","volume":"7","author":"Komaki","year":"2020","journal-title":"Ann Clin Transl Neurol"},{"key":"ref32","doi-asserted-by":"publisher","first-page":"982","DOI":"10.1001\/jamaneurol.2020.1264","volume":"77","author":"Clemens","year":"2020","journal-title":"JAMA Neurol"},{"key":"ref33","doi-asserted-by":"publisher","DOI":"10.1126\/scitranslmed.aan0713","volume":"10","author":"Komaki","year":"2018","journal-title":"Sci Transl Med"},{"key":"ref34","unstructured":"The Cooperative International Neuromuscular Research Group (CINRG). Available from: https:\/\/cinrgresearch.org\/duchenne-natural-history\/. Accessed November 15, 2021."},{"key":"ref35","unstructured":"NS PHARMA\u2019S VILTEPSOTM (viltolarsen) injection now FDA-approved in the U.S. for the treatment of Duchenne muscular dystrophy in patients amenable to exon 53 skipping therapy. NS Pharma News Release; 2020. Available from: https:\/\/www.nspharma.com\/pdfs\/Viltepso_Approval_Press_Release.pdf. Accessed November 15, 2021."},{"key":"ref36","unstructured":"ClinicalTrials.gov Exploratory Study of NS-065\/NCNP-01 in DMD. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02081625. Accessed November 15, 2021."},{"key":"ref37","unstructured":"ClinicalTrials.gov safety and dose finding study of NS-065\/NCNP-01 in boys with Duchenne Muscular Dystrophy (DMD). Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02740972. Accessed November 15, 2021."},{"key":"ref38","unstructured":"ClinicalTrials.gov Study to assess the efficacy and safety of viltolarsen in ambulant boys with DMD (RACER53). Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04060199. Accessed November 15, 2021."},{"key":"ref39","unstructured":"ClinicalTrials.gov Extension Study of NS-065\/NCNP-01 in boys with Duchenne Muscular Dystrophy (DMD). Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03167255. Accessed November 15, 2021."},{"key":"ref40","doi-asserted-by":"publisher","first-page":"4","DOI":"10.1016\/j.braindev.2015.05.014","volume":"38","author":"Matsuo","year":"2016","journal-title":"Brain Dev"},{"key":"ref41","doi-asserted-by":"publisher","first-page":"515","DOI":"10.1172\/JCI117693","volume":"95","author":"Takeshima","year":"1995","journal-title":"J Clin Invest"},{"key":"ref42","doi-asserted-by":"publisher","first-page":"2204","DOI":"10.1172\/JCI119757","volume":"100","author":"Shiga","year":"1997","journal-title":"J Clin Invest"},{"key":"ref43","doi-asserted-by":"publisher","first-page":"60","DOI":"10.1186\/1750-1172-8-60","volume":"8","author":"Nakamura","year":"2013","journal-title":"Orphanet J Rare Dis"},{"key":"ref44","unstructured":"Remudy. Available from: http:\/\/www.remudy.jp\/news\/2021\/09\/005992.html. Accessed November 15, 2021."},{"key":"ref45","unstructured":"Viltolarsen (viltepso) for Duchenne muscular dystrophy. Med Lett Drugs Ther. 2020;62(1609):167."},{"key":"ref46","doi-asserted-by":"publisher","first-page":"4098","DOI":"10.3390\/ijms20174098","volume":"20","author":"Meyers","year":"2019","journal-title":"Int J Mol Sci"}],"container-title":["Clinical Pharmacology: Advances and Applications"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.dovepress.com\/getfile.php?fileID=76798","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.dovepress.com\/getfile.php?fileID=76798","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,12,15]],"date-time":"2021-12-15T11:40:51Z","timestamp":1639568451000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.dovepress.com\/pharmacological-profile-of-viltolarsen-for-the-treatment-of-duchenne-m-peer-reviewed-fulltext-article-CPAA"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,12]]},"references-count":46,"URL":"https:\/\/doi.org\/10.2147\/cpaa.s288842","relation":{},"ISSN":["1179-1438"],"issn-type":[{"value":"1179-1438","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,12]]}}}