{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,2]],"date-time":"2026-11-02T19:02:34Z","timestamp":1793646154710,"version":"4.5.1"},"reference-count":32,"publisher":"American Medical Association (AMA)","issue":"8","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA Neurol"],"published-print":{"date-parts":[[2020,8,1]]},"DOI":"10.1001\/jamaneurol.2020.1264","type":"journal-article","created":{"date-parts":[[2020,5,26]],"date-time":"2020-05-26T11:02:02Z","timestamp":1590490922000},"page":"982","source":"Crossref","is-referenced-by-count":275,"title":["Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping"],"prefix":"10.1001","volume":"77","author":[{"given":"Paula R.","family":"Clemens","sequence":"first","affiliation":[{"name":"Department of Neurology, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania"},{"name":"Department of Veterans Affairs Medical Center, Pittsburgh, Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vamshi K.","family":"Rao","sequence":"additional","affiliation":[{"name":"Division of Neurology, Ann and Robert H. Lurie Children\u2019s Hospital of Chicago, Chicago, Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne M.","family":"Connolly","sequence":"additional","affiliation":[{"name":"Division of Neurology, Nationwide Children\u2019s Hospital, The Ohio State University College of Medicine, Columbus"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy D.","family":"Harper","sequence":"additional","affiliation":[{"name":"Children\u2019s Hospital of Richmond at Virginia Commonwealth University, Richmond"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean K.","family":"Mah","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of Calgary, Calgary, Alberta, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edward C.","family":"Smith","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Duke University Medical Center, Durham, North Carolina"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M.","family":"McDonald","sequence":"additional","affiliation":[{"name":"Department of Physical Medicine and Rehabilitation, Department of Pediatrics, UC Davis Health, University of California, Davis, Sacramento"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Craig M.","family":"Zaidman","sequence":"additional","affiliation":[{"name":"Department of Neurology, Washington University at St Louis, St Louis, Missouri"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lauren P.","family":"Morgenroth","sequence":"additional","affiliation":[{"name":"Graduate School of Public Health, University of Pittsburgh, Pittsburgh, Pennsylvania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hironori","family":"Osaki","sequence":"additional","affiliation":[{"name":"NS Pharma, Paramus, New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Youhei","family":"Satou","sequence":"additional","affiliation":[{"name":"NS Pharma, Paramus, New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Taishi","family":"Yamashita","sequence":"additional","affiliation":[{"name":"NS Pharma, Paramus, New Jersey"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric P.","family":"Hoffman","sequence":"additional","affiliation":[{"name":"AGADA BioSciences, Dalhousie University, Halifax, Nova Scotia, Canada"},{"name":"Department of Pharmaceutical Sciences, State University of New York at Binghamton"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"for the CINRG DNHS Investigators","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10","reference":[{"issue":"6","key":"noi200028r1","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","year":"1987","journal-title":"Cell"},{"issue":"14","key":"noi200028r2","doi-asserted-by":"crossref","first-page":"1209","DOI":"10.2217\/bmm-2019-0242","article-title":"Orthogonal analysis of dystrophin protein and mRNA as a surrogate outcome for drug development.","volume":"13","author":"Uaesoontrachoon","year":"2019","journal-title":"Biomark Med"},{"issue":"1","key":"noi200028r3","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/S0960-8966(99)00055-3","article-title":"Neuromuscular disorders in childhood: a descriptive epidemiological study from western Sweden.","volume":"10","author":"Darin","year":"2000","journal-title":"Neuromuscul Disord"},{"issue":"4","key":"noi200028r4","doi-asserted-by":"crossref","first-page":"395","DOI":"10.1002\/humu.22758","article-title":"The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations.","volume":"36","author":"Bladen","year":"2015","journal-title":"Hum Mutat"},{"issue":"21","key":"noi200028r5","doi-asserted-by":"crossref","first-page":"1363","DOI":"10.1056\/NEJM198805263182104","article-title":"Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne\u2019s or Becker\u2019s muscular dystrophy.","volume":"318","author":"Hoffman","year":"1988","journal-title":"N Engl J Med"},{"issue":"1","key":"noi200028r6","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1016\/S1474-4422(09)70271-6","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management.","volume":"9","author":"Bushby","year":"2010","journal-title":"Lancet Neurol"},{"issue":"1","key":"noi200028r7","first-page":"54","article-title":"Exploring the molecular basis for variability among patients with Becker muscular dystrophy: dystrophin gene and protein studies.","volume":"49","author":"Beggs","year":"1991","journal-title":"Am J Hum Genet"},{"issue":"12","key":"noi200028r8","doi-asserted-by":"crossref","first-page":"1048","DOI":"10.1212\/WNL.0000000000001950","article-title":"Prednisone\/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study.","volume":"85","author":"Bello","year":"2015","journal-title":"Neurology"},{"issue":"7","key":"noi200028r9","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1038\/s41582-019-0203-3","article-title":"Therapeutic developments for Duchenne muscular dystrophy.","volume":"15","author":"Verhaart","year":"2019","journal-title":"Nat Rev Neurol"},{"issue":"437","key":"noi200028r10","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aan0713","article-title":"Systemic administration of the antisense oligonucleotide NS-065\/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy.","volume":"10","author":"Komaki","year":"2018","journal-title":"Sci Transl Med"},{"key":"noi200028r11","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1016\/j.omtn.2018.09.017","article-title":"NS-065\/NCNP-01: an antisense oligonucleotide for potential treatment of exon 53 skipping in Duchenne muscular dystrophy.","volume":"13","author":"Watanabe","year":"2018","journal-title":"Mol Ther Nucleic Acids"},{"issue":"1","key":"noi200028r12","doi-asserted-by":"crossref","first-page":"32","DOI":"10.1002\/mus.23807","article-title":"The cooperative international neuromuscular research group Duchenne natural history study\u2014a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used.","volume":"48","author":"McDonald","year":"2013","journal-title":"Muscle Nerve"},{"issue":"10119","key":"noi200028r13","doi-asserted-by":"crossref","first-page":"451","DOI":"10.1016\/S0140-6736(17)32160-8","article-title":"Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study.","volume":"391","author":"McDonald","year":"2018","journal-title":"Lancet"},{"issue":"24","key":"noi200028r16","doi-asserted-by":"crossref","first-page":"e2146","DOI":"10.1212\/WNL.0000000000005680","article-title":"Eteplirsen treatment for Duchenne muscular dystrophy: exon skipping and dystrophin production.","volume":"90","author":"Charleston","year":"2018","journal-title":"Neurology"},{"issue":"15","key":"noi200028r17","article-title":"Golodirsen induces exon skipping leading to sarcolemmal dystrophin expression in Duchenne muscular dystrophy patients with mutations amenable to exon 53 skipping (S22.001).","volume":"90","author":"Muntoni","year":"2018","journal-title":"Neurology"},{"issue":"7","key":"noi200028r18","doi-asserted-by":"crossref","first-page":"747","DOI":"10.1136\/jnnp-2013-306350","article-title":"Dystrophin levels and clinical severity in Becker muscular dystrophy patients.","volume":"85","author":"van den Bergen","year":"2014","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"noi200028r19","doi-asserted-by":"crossref","first-page":"32439","DOI":"10.1038\/srep32439","article-title":"Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies.","volume":"6","author":"Bello","year":"2016","journal-title":"Sci Rep"},{"issue":"4","key":"noi200028r20","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0195850","article-title":"Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and Duchenne muscular dystrophy.","volume":"13","author":"Beekman","year":"2018","journal-title":"PLoS One"},{"issue":"1","key":"noi200028r21","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0083400","article-title":"6 Minute walk test in Duchenne MD patients with different mutations: 12 month changes.","volume":"9","author":"Pane","year":"2014","journal-title":"PLoS One"},{"issue":"9","key":"noi200028r22","doi-asserted-by":"crossref","first-page":"1193","DOI":"10.1002\/humu.23561","article-title":"DMD genotype correlations from the Duchenne Registry: endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation subtype.","volume":"39","author":"Wang","year":"2018","journal-title":"Hum Mutat"},{"key":"noi200028r25","doi-asserted-by":"crossref","first-page":"114","DOI":"10.3389\/fgene.2018.00114","article-title":"Combined therapies for Duchenne muscular dystrophy to optimize treatment efficacy.","volume":"9","author":"Cordova","year":"2018","journal-title":"Front Genet"},{"issue":"2","key":"noi200028r26","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1152\/physrev.00028.2001","article-title":"Function and genetics of dystrophin and dystrophin-related proteins in muscle.","volume":"82","author":"Blake","year":"2002","journal-title":"Physiol Rev"},{"issue":"1","key":"noi200028r27","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0052512","article-title":"24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy.","volume":"8","author":"Mazzone","year":"2013","journal-title":"PLoS One"},{"issue":"2","key":"noi200028r28","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1002\/ana.24555","article-title":"Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.","volume":"79","author":"Mendell","year":"2016","journal-title":"Ann Neurol"},{"issue":"5","key":"noi200028r29","doi-asserted-by":"crossref","first-page":"637","DOI":"10.1002\/ana.23982","article-title":"Eteplirsen for the treatment of Duchenne muscular dystrophy.","volume":"74","author":"Mendell","year":"2013","journal-title":"Ann Neurol"},{"issue":"1","key":"noi200028r30","doi-asserted-by":"crossref","first-page":"4","DOI":"10.1016\/j.nmd.2017.10.004","article-title":"A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy.","volume":"28","author":"Goemans","year":"2018","journal-title":"Neuromuscul Disord"},{"key":"noi200028r14","unstructured":"Extension study of NS-065\/NCNP-01 in boys with Duchenne muscular dystrophy (DMD). ClinicalTrials.gov identifier: NCT03167255. Updated March 21, 2019. Accessed January 6, 2020. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03167255"},{"key":"noi200028r15","unstructured":"US Food and Drug Administration. Table of surrogate endpoints that were the basis of drug approval or licensure. Accessed December 6, 2019. https:\/\/www.fda.gov\/drugs\/development-resources\/table-surrogate-endpoints-were-basis-drug-approval-or-licensure"},{"key":"noi200028r23","unstructured":"US Food and Drug Administration. Duchenne muscular dystrophy and related dystrophinopathies: developing drugs for treatment guidance for industry. Accessed December 6, 2019. https:\/\/www.fda.gov\/regulatory-information\/search-fda-guidance-documents\/duchenne-muscular-dystrophy-and-related-dystrophinopathies-developing-drugs-treatment-guidance"},{"key":"noi200028r24","unstructured":"Institute for Clinical and Economic Review. Deflazacort, eteplirsen, and golodirsen for Duchenne muscular dystrophy: effectiveness and value. Accessed December 6, 2019. https:\/\/icer-review.org\/wp-content\/uploads\/2018\/12\/ICER_DMD_Draft_Scope_011119-1.pdf"},{"key":"noi200028r31","unstructured":"BioMarin announces withdrawal of market authorization application for Kyndrisa\u2122 (drisapersen) in Europe. News release. Globe Newswire; May 31, 2016. Accessed December 6, 2019. https:\/\/www.globenewswire.com\/news-release\/2016\/05\/31\/844933\/0\/en\/BioMarin-Announces-Withdrawal-of-Market-Authorization-Application-for-Kyndrisa-drisapersen-in-Europe.html"},{"key":"noi200028r32","unstructured":"Phase I\/II study of SRP-4053 in DMD patients. ClinicalTrials.gov identifier: NCT02310906. Updated June 12, 2019. Accessed December 6, 2019. https:\/\/clinicaltrials.gov\/ct2\/show\/NCT02310906"}],"container-title":["JAMA Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/jamanetwork.com\/journals\/jamaneurology\/articlepdf\/2766519\/jamaneurology_clemens_2020_oi_200028_1596744498.28895.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,8,10]],"date-time":"2020-08-10T11:15:59Z","timestamp":1597058159000},"score":1,"resource":{"primary":{"URL":"https:\/\/jamanetwork.com\/journals\/jamaneurology\/fullarticle\/2766519"}},"subtitle":["A Phase 2 Randomized Clinical Trial"],"short-title":[],"issued":{"date-parts":[[2020,8,1]]},"references-count":32,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2020,8,1]]}},"URL":"https:\/\/doi.org\/10.1001\/jamaneurol.2020.1264","relation":{},"ISSN":["2168-6149"],"issn-type":[{"value":"2168-6149","type":"print"}],"subject":[],"published":{"date-parts":[[2020,8,1]]}}}