{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,29]],"date-time":"2026-03-29T07:58:11Z","timestamp":1774771091893,"version":"3.50.1"},"reference-count":20,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2019,4,2]],"date-time":"2019-04-02T00:00:00Z","timestamp":1554163200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Orphanet J Rare Dis"],"published-print":{"date-parts":[[2019,12]]},"DOI":"10.1186\/s13023-018-0975-3","type":"journal-article","created":{"date-parts":[[2019,4,2]],"date-time":"2019-04-02T16:04:14Z","timestamp":1554221054000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Incomplete description of the current body of evidence of the health economics of Duchenne muscular dystrophy"],"prefix":"10.1186","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5758-7151","authenticated-orcid":false,"given":"Erik","family":"Landfeldt","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hanns","family":"Lochm\u00fcller","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Peter","family":"Lindgren","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2019,4,2]]},"reference":[{"issue":"1","key":"975_CR1","doi-asserted-by":"publisher","first-page":"79","DOI":"10.1186\/s13023-017-0631-3","volume":"12","author":"S Ryder","year":"2017","unstructured":"Ryder S, Leadley RM, Armstrong N, et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review. Orphanet J Rare Dis. 2017;12(1):79.","journal-title":"Orphanet J Rare Dis"},{"issue":"5","key":"975_CR2","doi-asserted-by":"publisher","first-page":"508","DOI":"10.1111\/dmcn.12938","volume":"58","author":"E Landfeldt","year":"2016","unstructured":"Landfeldt E, Lindgren P, Bell C, et al. Health-related quality of life in patients with Duchenne muscular dystrophy: a multi-national, cross-sectional study. Dev Med Child Neurol. 2016;58(5):508\u201315.","journal-title":"Dev Med Child Neurol"},{"issue":"2","key":"975_CR3","doi-asserted-by":"publisher","first-page":"249","DOI":"10.1007\/s40273-016-0461-5","volume":"35","author":"E Landfeldt","year":"2017","unstructured":"Landfeldt E, Alfredsson L, Straub V, et al. Economic evaluation in Duchenne muscular dystrophy: model frameworks for cost-effectiveness analysis. PharmacoEconomics. 2017;35(2):249\u201358.","journal-title":"PharmacoEconomics"},{"issue":"6","key":"975_CR4","doi-asserted-by":"publisher","first-page":"877","DOI":"10.1002\/mus.24965","volume":"53","author":"LJ Teoh","year":"2016","unstructured":"Teoh LJ, Geelhoed EA, Bayley K, et al. Health care utilization and costs for children and adults with duchenne muscular dystrophy. Muscle Nerve. 2016;53(6):877\u201384.","journal-title":"Muscle Nerve"},{"issue":"4","key":"975_CR5","doi-asserted-by":"publisher","first-page":"709","DOI":"10.1007\/s00415-017-8406-2","volume":"264","author":"C Otto","year":"2017","unstructured":"Otto C, Steffensen BF, H\u00f8jberg AL, et al. Predictors of health-related quality of life in boys with Duchenne muscular dystrophy from six European countries. J Neurol. 2017;264(4):709\u201323.","journal-title":"J Neurol"},{"issue":"14","key":"975_CR6","doi-asserted-by":"publisher","first-page":"1408","DOI":"10.1080\/09638288.2016.1196398","volume":"39","author":"YJ Lue","year":"2017","unstructured":"Lue YJ, Chen SS, Lu YM, et al. Quality of life of patients with Duchenne muscular dystrophy: from adolescence to young men. Disabil Rehabil. 2017;39(14):1408\u201313.","journal-title":"Disabil Rehabil"},{"issue":"7","key":"975_CR7","doi-asserted-by":"publisher","first-page":"423","DOI":"10.1016\/j.nmd.2016.05.004","volume":"26","author":"G Zamani","year":"2016","unstructured":"Zamani G, Heidari M, Azizi Malamiri R, et al. The quality of life in boys with Duchenne muscular dystrophy. Neuromuscul Disord. 2016;26(7):423\u20137.","journal-title":"Neuromuscul Disord"},{"issue":"Suppl 1","key":"975_CR8","doi-asserted-by":"publisher","first-page":"19","DOI":"10.1007\/s10198-016-0782-5","volume":"17","author":"M Cavazza","year":"2016","unstructured":"Cavazza M, Kodra Y, Armeni P, et al. Social\/economic costs and health-related quality of life in patients with Duchenne muscular dystrophy in Europe. Eur J Health Econ. 2016;17(Suppl 1):19\u201329.","journal-title":"Eur J Health Econ"},{"issue":"3","key":"975_CR9","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1016\/j.nmd.2016.01.003","volume":"26","author":"S Messina","year":"2016","unstructured":"Messina S, Vita GL, Sframeli M, et al. Health-related quality of life and functional changes in DMD: a 12-month longitudinal cohort study. Neuromuscul Disord. 2016;26(3):189\u201396.","journal-title":"Neuromuscul Disord"},{"key":"975_CR10","doi-asserted-by":"publisher","first-page":"97","DOI":"10.1097\/CND.0b013e3181c5053b","volume":"11","author":"SE Davis","year":"2010","unstructured":"Davis SE, Hynan LS, Limbers CA, et al. The PedsQL in pediatric patients with Duchenne muscular dystrophy: feasibility, reliability, and validity of the pediatric quality of life inventory neuromuscular module and generic Core scales. J Clin Neuromuscul Dis. 2010;11:97\u2013109.","journal-title":"J Clin Neuromuscul Dis"},{"key":"975_CR11","doi-asserted-by":"publisher","first-page":"1188","DOI":"10.1177\/0883073809357624","volume":"25","author":"P Bray","year":"2010","unstructured":"Bray P, Bundy AC, Ryan MM, North KN, Everett A. Health-related quality of life in boys with Duchenne muscular dystrophy: agreement between parents and their sons. J Child Neurol. 2010;25:1188\u201394.","journal-title":"J Child Neurol"},{"key":"975_CR12","doi-asserted-by":"publisher","first-page":"557","DOI":"10.1111\/j.1440-1754.2011.02022.x","volume":"47","author":"P Bray","year":"2011","unstructured":"Bray P, Bundy AC, Ryan MM, et al. Health status of boys with Duchenne muscular dystrophy: a parent\u2019s perspective. J Paediatr Child Health. 2011;47:557\u201362.","journal-title":"J Paediatr Child Health"},{"key":"975_CR13","doi-asserted-by":"publisher","first-page":"1032","DOI":"10.1164\/rccm.200503-322OC","volume":"172","author":"M Kohler","year":"2005","unstructured":"Kohler M, Clarenbach CF, Boni L, et al. Quality of life, physical disability, and respiratory impairment in Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2005;172:1032\u20136.","journal-title":"Am J Respir Crit Care Med"},{"key":"975_CR14","doi-asserted-by":"publisher","first-page":"257","DOI":"10.1055\/s-0033-1347935","volume":"44","author":"S Elsenbruch","year":"2013","unstructured":"Elsenbruch S, Schmid J, Lutz S, et al. Self-reported quality of life and depressive symptoms in children, adolescents, and adults with Duchenne muscular dystrophy: a cross-sectional survey study. Neuropediatrics. 2013;44:257\u201364.","journal-title":"Neuropediatrics"},{"key":"975_CR15","doi-asserted-by":"publisher","first-page":"47","DOI":"10.1186\/1477-7525-11-47","volume":"11","author":"J Hu","year":"2013","unstructured":"Hu J, Jiang L, Hong S, et al. Reliability and validity of the Chinese version of the pediatric quality of life InventoryTM (PedsQLTM) 3.0 neuromuscular module in children with Duchenne muscular dystrophy. Health Qual Life Outcomes. 2013;11:47.","journal-title":"Health Qual Life Outcomes"},{"key":"975_CR16","doi-asserted-by":"publisher","first-page":"1130","DOI":"10.1177\/0883073810371509","volume":"25","author":"CM McDonald","year":"2010","unstructured":"McDonald CM, McDonald DA, Bagley A, et al. Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy. J Child Neurol. 2010;25:1130\u201344.","journal-title":"J Child Neurol"},{"key":"975_CR17","doi-asserted-by":"publisher","first-page":"431","DOI":"10.1002\/mus.23942","volume":"49","author":"J Larkindale","year":"2014","unstructured":"Larkindale J, Yang W, Hogan PF, et al. Cost of illness for neuromuscular diseases in the United States. Muscle Nerve. 2014;49:431\u20138.","journal-title":"Muscle Nerve"},{"key":"975_CR18","doi-asserted-by":"publisher","first-page":"529","DOI":"10.1212\/WNL.0000000000000669","volume":"83","author":"E Landfeldt","year":"2014","unstructured":"Landfeldt E, Lindgren P, Bell C, et al. The burden of Duchenne muscular dystrophy: an international, cross-sectional study. Neurology. 2014;83:529\u201336.","journal-title":"Neurology"},{"key":"975_CR19","doi-asserted-by":"publisher","first-page":"e1559","DOI":"10.1542\/peds.2012-0858","volume":"130","author":"K Uzark","year":"2012","unstructured":"Uzark K, King E, Cripe L, et al. Health-related quality of life in children and adolescents with Duchenne muscular dystrophy. Pediatrics. 2012;130:e1559\u201366.","journal-title":"Pediatrics"},{"issue":"7","key":"975_CR20","doi-asserted-by":"publisher","first-page":"1945","DOI":"10.1007\/s11136-014-0642-7","volume":"23","author":"Y Lim","year":"2014","unstructured":"Lim Y, Velozo C, Bendixen RM. The level of agreement between child self-reports and parent proxy-reports of health-related quality of life in boys with Duchenne muscular dystrophy. Qual Life Res. 2014;23(7):1945\u201352.","journal-title":"Qual Life Res"}],"container-title":["Orphanet Journal of Rare Diseases"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1186\/s13023-018-0975-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1186\/s13023-018-0975-3\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1186\/s13023-018-0975-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,3,31]],"date-time":"2020-03-31T23:21:00Z","timestamp":1585696860000},"score":1,"resource":{"primary":{"URL":"https:\/\/ojrd.biomedcentral.com\/articles\/10.1186\/s13023-018-0975-3"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,4,2]]},"references-count":20,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2019,12]]}},"alternative-id":["975"],"URL":"https:\/\/doi.org\/10.1186\/s13023-018-0975-3","relation":{},"ISSN":["1750-1172"],"issn-type":[{"value":"1750-1172","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,4,2]]},"assertion":[{"value":"3 October 2017","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"10 December 2018","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"2 April 2019","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Not applicable.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"The authors declare that they have no competing interests.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}},{"value":"Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Publisher\u2019s Note"}}],"article-number":"75"}}