{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T01:46:33Z","timestamp":1792028793659,"version":"4.4.0"},"reference-count":45,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2026,11,1]],"date-time":"2026-11-01T00:00:00Z","timestamp":1793491200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,11,1]],"date-time":"2026-11-01T00:00:00Z","timestamp":1793491200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T00:00:00Z","timestamp":1772150400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100006506","name":"Minist\u00e9rio da Sa\u00fade","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100006506","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["valuehealthregionalissues.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Value in Health Regional Issues"],"published-print":{"date-parts":[[2026,11]]},"DOI":"10.1016\/j.vhri.2026.101623","type":"journal-article","created":{"date-parts":[[2026,3,26]],"date-time":"2026-03-26T12:31:19Z","timestamp":1774528279000},"page":"101623","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"special_numbering":"C","title":["Cost-Utility Analysis of the Treatment With Ataluren Plus Standard of Care Compared With Standard of Care Alone in Patients With Duchenne Muscular Dystrophy in Brazil"],"prefix":"10.1016","volume":"56","author":[{"given":"Eliane W.","family":"Roesch","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ver\u00f4nica","family":"Colpani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Grace Anne A.","family":"D\u00f3ria","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kl\u00e9bya Hellen D.","family":"de Oliveira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catia","family":"Oliveira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henry M.","family":"Peixoto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana Fl\u00e1via de M.","family":"Oliveira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"1","key":"10.1016\/j.vhri.2026.101623_bib1","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1038\/s41572-021-00248-3","article-title":"Duchenne muscular dystrophy","volume":"7","author":"Duan","year":"2021","journal-title":"Nat Rev Dis Primer"},{"issue":"1","key":"10.1016\/j.vhri.2026.101623_bib2","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1016\/0960-8966(91)90039-U","article-title":"Population frequencies of inherited neuromuscular diseases\u2014a world survey","volume":"1","author":"Emery","year":"1991","journal-title":"Neuromuscul Disord"},{"issue":"9","key":"10.1016\/j.vhri.2026.101623_bib3","doi-asserted-by":"crossref","first-page":"69591","DOI":"10.34117\/bjdv6n9-416","article-title":"Epidemiology of Duchenne muscular dystrophy in Cear\u00e1","volume":"6","author":"Teixeira","year":"2020","journal-title":"Braz J Dev"},{"issue":"2","key":"10.1016\/j.vhri.2026.101623_bib4","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1111\/cge.12975","article-title":"Genetic profile of Brazilian patients with dystrophinopathies","volume":"92","author":"De Almeida","year":"2017","journal-title":"Clin Genet"},{"issue":"23","key":"10.1016\/j.vhri.2026.101623_bib5","doi-asserted-by":"crossref","first-page":"e2304","DOI":"10.1212\/WNL.0000000000012910","article-title":"Life expectancy in Duchenne muscular dystrophy: reproduced individual patient data meta-analysis","volume":"97","author":"Broomfield","year":"2021","journal-title":"Neurology"},{"issue":"7","key":"10.1016\/j.vhri.2026.101623_bib6","doi-asserted-by":"crossref","first-page":"643","DOI":"10.1007\/s10654-020-00613-8","article-title":"Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis","volume":"35","author":"Landfeldt","year":"2020","journal-title":"Eur J Epidemiol"},{"issue":"6","key":"10.1016\/j.vhri.2026.101623_bib7","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1212\/WNL.0000000000000669","article-title":"The burden of Duchenne muscular dystrophy: an international, cross-sectional study","volume":"83","author":"Landfeldt","year":"2014","journal-title":"Neurology"},{"issue":"1","key":"10.1016\/j.vhri.2026.101623_bib8","doi-asserted-by":"crossref","first-page":"159","DOI":"10.1186\/s13023-023-02767-6","article-title":"Estimated costs for Duchenne muscular dystrophy care in Brazil","volume":"18","author":"Schneider","year":"2023","journal-title":"Orphanet J Rare Dis"},{"issue":"01","key":"10.1016\/j.vhri.2026.101623_bib9","doi-asserted-by":"crossref","first-page":"081","DOI":"10.1055\/s-0043-1761466","article-title":"Update of the Brazilian consensus recommendations on Duchenne muscular dystrophy","volume":"81","author":"Araujo","year":"2023","journal-title":"Arq Neuropsiquiatr"},{"issue":"3","key":"10.1016\/j.vhri.2026.101623_bib10","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1016\/S1474-4422(18)30024-3","article-title":"Diagnosis and management of Duchenne muscular dystrophy, part 1: Diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management","volume":"17","author":"Birnkrant","year":"2018","journal-title":"Lancet Neurol"},{"issue":"1","key":"10.1016\/j.vhri.2026.101623_bib11","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":"9","key":"10.1016\/j.vhri.2026.101623_bib12","doi-asserted-by":"crossref","first-page":"1251","DOI":"10.7326\/M23-1073","article-title":"The regulatory repercussions of approving muscular dystrophy medications on the basis of limited evidence","volume":"176","author":"Bendicksen","year":"2023","journal-title":"Ann Intern Med"},{"issue":"9","key":"10.1016\/j.vhri.2026.101623_bib13","doi-asserted-by":"crossref","first-page":"814","DOI":"10.1016\/S1474-4422(22)00125-9","article-title":"Emerging therapies for Duchenne muscular dystrophy","volume":"21","author":"Markati","year":"2022","journal-title":"Lancet Neurol"},{"issue":"4","key":"10.1016\/j.vhri.2026.101623_bib14","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1002\/mus.24332","article-title":"Ataluren treatment of patients with nonsense mutation dystrophinopathy","volume":"50","author":"Bushby","year":"2014","journal-title":"Muscle Nerve"},{"issue":"10101","key":"10.1016\/j.vhri.2026.101623_bib15","doi-asserted-by":"crossref","first-page":"1489","DOI":"10.1016\/S0140-6736(17)31611-2","article-title":"Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial","volume":"390","author":"McDonald","year":"2017","journal-title":"Lancet"},{"issue":"3","key":"10.1016\/j.vhri.2026.101623_bib16","doi-asserted-by":"crossref","first-page":"139","DOI":"10.2217\/cer-2021-0196","article-title":"Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients","volume":"11","author":"McDonald","year":"2022","journal-title":"J Comp Eff Res"},{"issue":"8","key":"10.1016\/j.vhri.2026.101623_bib17","doi-asserted-by":"crossref","first-page":"3896","DOI":"10.1007\/s00415-023-11687-1","article-title":"Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis","volume":"270","author":"Mercuri","year":"2023","journal-title":"J Neurol"},{"issue":"1","key":"10.1016\/j.vhri.2026.101623_bib18","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1016\/j.jval.2021.10.008","article-title":"Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 explanation and elaboration: a report of the ISPOR Cheers II good practices task force","volume":"25","author":"Husereau","year":"2022","journal-title":"Value Health"},{"issue":"2","key":"10.1016\/j.vhri.2026.101623_bib19","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1007\/s40273-016-0461-5","article-title":"Economic evaluation in Duchenne muscular dystrophy: model frameworks for cost-effectiveness analysis","volume":"35","author":"Landfeldt","year":"2017","journal-title":"Pharmacoeconomics"},{"key":"10.1016\/j.vhri.2026.101623_bib21","series-title":"Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene","year":"2023"},{"key":"10.1016\/j.vhri.2026.101623_bib22","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1016\/j.vhri.2023.08.004","article-title":"Early cost-utility analysis of ataluren and Eteplirsen in the treatment of Duchenne muscular dystrophy in Egypt","volume":"38","author":"Shehata","year":"2023","journal-title":"Value Health Reg Issues"},{"issue":"7","key":"10.1016\/j.vhri.2026.101623_bib23","doi-asserted-by":"crossref","first-page":"481","DOI":"10.1590\/0004-282x20180062","article-title":"Brazilian consensus on Duchenne muscular dystrophy. Part 2: rehabilitation and systemic care","volume":"76","author":"Araujo","year":"2018","journal-title":"Arq Neuropsiquiatr"},{"key":"10.1016\/j.vhri.2026.101623_bib24","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1016\/j.vhri.2022.08.006","article-title":"Estimating societal cost of illness and patients\u2019 quality of life of Duchenne muscular dystrophy in Egypt","volume":"33","author":"Shehata","year":"2023","journal-title":"Value Health Reg Issues"},{"issue":"1","key":"10.1016\/j.vhri.2026.101623_bib25","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1007\/s40273-015-0337-0","article-title":"Myths and misconceptions of within-cycle correction: a guide for modelers and decision makers","volume":"34","author":"Elbasha","year":"2016","journal-title":"Pharmacoeconomics"},{"key":"10.1016\/j.vhri.2026.101623_bib26","article-title":"Uso de Limiares de custo-efetividade na Decis\u00f5es em sa\u00fade: Recomenda\u00e7\u00f5es da comiss\u00e3o nacional de incorpora\u00e7\u00e3o de tecnologias no SUS. Brazil","year":"2022","journal-title":"Minist\u00e9rio da Sa\u00fade"},{"issue":"2","key":"10.1016\/j.vhri.2026.101623_bib27","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1590\/S0103-51502010000200006","article-title":"Correla\u00e7\u00e3o da massa e porcentagem de gordura com a idade na distrofia muscular de Duchenne","volume":"23","author":"Caromano","year":"2010","journal-title":"Fisioter Mov"},{"key":"10.1016\/j.vhri.2026.101623_bib31","series-title":"World Development Indicators","year":"2023"},{"issue":"3","key":"10.1016\/j.vhri.2026.101623_bib33","doi-asserted-by":"crossref","first-page":"593","DOI":"10.1007\/s11136-019-02355-x","article-title":"Characterizing health state utilities associated with Duchenne muscular dystrophy: a systematic review","volume":"29","author":"Szabo","year":"2020","journal-title":"Qual Life Res"},{"issue":"1","key":"10.1016\/j.vhri.2026.101623_bib34","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1186\/s41687-023-00671-y","article-title":"Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L","volume":"7","author":"Audhya","year":"2023","journal-title":"J Patient-Rep Outcomes"},{"issue":"1","key":"10.1016\/j.vhri.2026.101623_bib35","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1186\/s12883-024-03570-x","article-title":"The role of ataluren in the treatment of ambulatory and non-ambulatory children with nonsense mutations Duchenne muscular dystrophy - a consensus derived using a modified Delphi methodology in Eastern Europe, Greece, Israel and Sweden","volume":"24","author":"Golli","year":"2024","journal-title":"BMC Neurol"},{"key":"10.1016\/j.vhri.2026.101623_bib36","doi-asserted-by":"crossref","DOI":"10.1016\/j.healthpol.2024.105044","article-title":"Lessons learned from the Canadian Fabry Disease Initiative for future risk-sharing and managed access agreements for pharmaceutical and advanced therapies in Canada","volume":"143","author":"Douglas","year":"2024","journal-title":"Health Policy"},{"issue":"6","key":"10.1016\/j.vhri.2026.101623_bib37","doi-asserted-by":"crossref","DOI":"10.1242\/dmm.050623","article-title":"Improving access to gene therapy for rare diseases","volume":"17","author":"Fox","year":"2024","journal-title":"Dis Model Mech"},{"issue":"10","key":"10.1016\/j.vhri.2026.101623_bib38","doi-asserted-by":"crossref","first-page":"779","DOI":"10.2471\/BLT.15.163519","article-title":"Negotiating prices of drugs for rare diseases","volume":"94","author":"Henrard","year":"2016","journal-title":"Bull World Health Organ"},{"issue":"Suppl 1","key":"10.1016\/j.vhri.2026.101623_bib39","first-page":"101","article-title":"Risk Sharing Agreement: a pilot project in the Brazilian Unified Health System","volume":"14","author":"Vianna","year":"2022","journal-title":"J Bras Econ Sa\u00fade"},{"issue":"3","key":"10.1016\/j.vhri.2026.101623_bib40","first-page":"14","article-title":"Avalia\u00e7\u00e3o de tecnologias para doen\u00e7as raras e judicializa\u00e7\u00e3o: rela\u00e7\u00e3o entre as demandas judiciais e as a\u00e7\u00f5es da Comiss\u00e3o Nacional de Incorpora\u00e7\u00e3o de Tecnologias no Sistema \u00danico de Sa\u00fade (CONITEC)","volume":"5","author":"Beutel","year":"2022","journal-title":"J Assist Farm Farm"},{"issue":"11","key":"10.1016\/j.vhri.2026.101623_bib41","doi-asserted-by":"crossref","first-page":"5547","DOI":"10.1590\/1413-812320212611.26722020","article-title":"Incorpora\u00e7\u00f5es de medicamentos para doen\u00e7as raras no Brasil: \u00e9 poss\u00edvel acesso integral a estes pacientes?","volume":"26","author":"Biglia","year":"2021","journal-title":"Ci\u00eanc Sa\u00fade Colet"},{"key":"10.1016\/j.vhri.2026.101623_bib42","doi-asserted-by":"crossref","first-page":"1","DOI":"10.11606\/s1518-8787.2023057004579","article-title":"Judicializa\u00e7\u00e3o e direito \u00e0 sa\u00fade no Brasil: uma trajet\u00f3ria de encontros e desencontros","volume":"57","author":"Vieira","year":"2023","journal-title":"Rev Sa\u00fade Publ"},{"key":"10.1016\/j.vhri.2026.101623_bib43","article-title":"Advancing rare disease policy in Latin America: a call to action","volume":"18","author":"Wainstock","year":"2023","journal-title":"Lancet Reg Health Am"},{"issue":"12","key":"10.1016\/j.vhri.2026.101623_bib44","doi-asserted-by":"crossref","first-page":"1641","DOI":"10.1016\/j.jval.2024.07.008","article-title":"Discriminatory properties of the quality-adjusted life year based cost-effectiveness analyses for patients with disabilities: a Duchenne muscular dystrophy case study","volume":"27","author":"Klimchak","year":"2024","journal-title":"Value Health"},{"issue":"12","key":"10.1016\/j.vhri.2026.101623_bib45","doi-asserted-by":"crossref","first-page":"1635","DOI":"10.1016\/j.jval.2024.10.3803","article-title":"Rare diseases: economic evaluation and policy considerations","volume":"27","author":"Rittenhouse","year":"2024","journal-title":"Value Health"},{"issue":"12","key":"10.1016\/j.vhri.2026.101623_bib46","doi-asserted-by":"crossref","first-page":"1648","DOI":"10.1016\/j.jval.2024.06.014","article-title":"Three pillars of support for orphan drug programs: individual valuations, societal valuations, and anonymous altruism","volume":"27","author":"Lakdawalla","year":"2024","journal-title":"Value Health"},{"issue":"3","key":"10.1016\/j.vhri.2026.101623_bib47","doi-asserted-by":"crossref","first-page":"496","DOI":"10.1590\/0102-311x00213813","article-title":"Economic evaluation in the context of rare diseases: is it possible?","volume":"31","author":"Silva","year":"2015","journal-title":"Cad Sa\u00fade Publ"},{"key":"10.1016\/j.vhri.2026.101623_bib48","article-title":"Societal preferences in health technology assessments for rare diseases and orphan drugs: a systematic literature review of new analytic approaches","volume":"44","author":"V\u00e1squez","year":"2024","journal-title":"Value Health Reg Issues"},{"issue":"1","key":"10.1016\/j.vhri.2026.101623_bib49","doi-asserted-by":"crossref","first-page":"332","DOI":"10.1038\/s41591-024-03304-z","article-title":"AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial","volume":"31","author":"Mendell","year":"2025","journal-title":"Nat Med"},{"issue":"4","key":"10.1016\/j.vhri.2026.101623_bib50","doi-asserted-by":"crossref","first-page":"393","DOI":"10.1016\/S1474-4422(24)00036-X","article-title":"Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial","volume":"23","author":"Mercuri","year":"2024","journal-title":"Lancet Neurol"}],"container-title":["Value in Health Regional Issues"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2212109926000385?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2212109926000385?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T01:31:10Z","timestamp":1792027870000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2212109926000385"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,11]]},"references-count":45,"alternative-id":["S2212109926000385"],"URL":"https:\/\/doi.org\/10.1016\/j.vhri.2026.101623","relation":{},"ISSN":["2212-1099"],"issn-type":[{"value":"2212-1099","type":"print"}],"subject":[],"published":{"date-parts":[[2026,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Cost-Utility Analysis of the Treatment With Ataluren Plus Standard of Care Compared With Standard of Care Alone in Patients With Duchenne Muscular Dystrophy in Brazil","name":"articletitle","label":"Article Title"},{"value":"Value in Health Regional Issues","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.vhri.2026.101623","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Authors. Published by Elsevier Inc. on behalf of International Society for Pharmacoeconomics and Outcomes Research, Inc.","name":"copyright","label":"Copyright"}],"article-number":"101623"}}