{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,29]],"date-time":"2026-08-29T12:17:09Z","timestamp":1788005829509,"version":"build-2784847793"},"reference-count":19,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2021,5,1]],"date-time":"2021-05-01T00:00:00Z","timestamp":1619827200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2021,5,1]],"date-time":"2021-05-01T00:00:00Z","timestamp":1619827200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2021,3,1]],"date-time":"2021-03-01T00:00:00Z","timestamp":1614556800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2021,5]]},"DOI":"10.1016\/j.ymgme.2021.02.006","type":"journal-article","created":{"date-parts":[[2021,2,27]],"date-time":"2021-02-27T07:12:10Z","timestamp":1614409930000},"page":"94-99","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":18,"title":["Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study"],"prefix":"10.1016","volume":"133","author":[{"given":"Dafne D.G.","family":"Horovitz","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Em\u00edlia K.E.A.","family":"Le\u00e3o","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erlane M.","family":"Ribeiro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana M.","family":"Martins","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anneliese L.","family":"Barth","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jo\u00e3o I.C.F.","family":"Neri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcelo","family":"Kerstenetzky","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana C.M.","family":"Siqueira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bethania F.R.","family":"Ribeiro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chong A.","family":"Kim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francisca C.","family":"Santos","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jos\u00e9 F.S.","family":"Franco","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leniza C.L.","family":"Lichtvan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liane R.","family":"Giuliani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Do C.S.","family":"Rodrigues","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renata C.F.","family":"Bonatti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tha\u00eds B.","family":"Teixeira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra","family":"Gon\u00e7alves","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles M.","family":"Louren\u00e7o","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ane S.S.","family":"Pereira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angelina X.","family":"Acosta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymgme.2021.02.006_bb0005","series-title":"Metab. Mol. Bases Inherit. Dis.","first-page":"3421","article-title":"The mucopolysaccharidoses","author":"Neufeld","year":"2001"},{"key":"10.1016\/j.ymgme.2021.02.006_bb0010","doi-asserted-by":"crossref","first-page":"208","DOI":"10.1111\/j.1399-0004.2004.00277.x","article-title":"Clinical and biochemical study of 28 patients with mucopolysaccharidosis type VI","volume":"66","author":"Azevedo","year":"2004","journal-title":"Clin Genet."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0015","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1002\/ajmg.a.30579","article-title":"Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)","volume":"134A","author":"Swiedler","year":"2005","journal-title":"Am. J. Med. Genet. A"},{"key":"10.1016\/j.ymgme.2021.02.006_bb0020","doi-asserted-by":"crossref","first-page":"1153","DOI":"10.1038\/s10038-019-0662-9","article-title":"Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future","volume":"64","author":"Chen","year":"2019","journal-title":"J. Hum. Genet."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0025","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1111\/j.1399-0004.2009.01324.x","article-title":"Enzyme replacement therapy for mucopolysaccharidosis VI from 8 weeks of age\u2013a sibling control study","volume":"77","author":"McGill","year":"2010","journal-title":"Clin. Genet."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0030","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1016\/j.ymgme.2013.02.014","article-title":"Enzyme replacement therapy with galsulfase in 34 children younger than five years of age with MPS VI","volume":"109","author":"Horovitz","year":"2013","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0035","first-page":"1","article-title":"Growth charts for individuals with Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome)","volume":"18","author":"Quartel","year":"2015","journal-title":"JIMD Rep."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0040","doi-asserted-by":"crossref","first-page":"1953","DOI":"10.1002\/ajmg.a.36584","article-title":"Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study","volume":"164A","author":"Giugliani","year":"2014","journal-title":"Am. J. Med. Genet. A"},{"key":"10.1016\/j.ymgme.2021.02.006_bb0045","doi-asserted-by":"crossref","DOI":"10.1177\/2326409818755800","article-title":"Long-term galsulfase treatment associated with improved survival of patients with mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): 15-year follow-up from the survey study","volume":"6","author":"Quartel","year":"2018","journal-title":"J. Inborn Errors Metab. Screen."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0050","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1016\/j.ymgme.2020.02.006","article-title":"Urinary glycosaminoglycans as a potential biomarker for evaluating treatment efficacy in subjects with mucopolysaccharidoses","volume":"130","author":"Kakkis","year":"2020","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0055","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1007\/s10545-013-9654-7","article-title":"Galsulfase (Naglazyme\u00ae) therapy in infants with mucopolysaccharidosis VI","volume":"37","author":"Harmatz","year":"2014","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0060","doi-asserted-by":"crossref","first-page":"597","DOI":"10.1016\/j.ymgme.2011.08.029","article-title":"Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI","volume":"104","author":"Furujo","year":"2011","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0065","first-page":"89","article-title":"Enzyme replacement therapy for mucopolysaccharidosis VI: growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase","volume":"3","author":"Decker","year":"2010","journal-title":"J. Pediatr. Rehabil. Med."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0070","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1016\/j.ymgmr.2015.09.002","article-title":"Mucopolysaccharidosis type VI on enzyme replacement therapy since infancy: Six years follow-up of four children","volume":"5","author":"Horovitz","year":"2015","journal-title":"Mol. Genet. Metab. Reports"},{"key":"10.1016\/j.ymgme.2021.02.006_bb0075","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.1007\/s10545-011-9359-8","article-title":"Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management","volume":"34","author":"Braunlin","year":"2011","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0080","doi-asserted-by":"crossref","first-page":"519","DOI":"10.1002\/jimd.12079","article-title":"Enzyme replacement therapy outcomes across the disease spectrum: findings from the mucopolysaccharidosis VI Clinical Surveillance Program","volume":"42","author":"Harmatz","year":"2019","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0085","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1186\/1750-1172-5-5","article-title":"Mucopolysaccharidosis VI","volume":"5","author":"Valayannopoulos","year":"2010","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0090","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1186\/s13052-018-0564-z","article-title":"New treatments for the mucopolysaccharidoses: from pathophysiology to therapy","volume":"44","author":"Fecarotta","year":"2018","journal-title":"Ital. J. Pediatr."},{"key":"10.1016\/j.ymgme.2021.02.006_bb0095","doi-asserted-by":"crossref","DOI":"10.3390\/ijms21072515","article-title":"Pathogenesis of mucopolysaccharidoses, an update","volume":"21","author":"Fecarotta","year":"2020","journal-title":"Int. J. Mol. Sci."}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719221000548?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719221000548?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,21]],"date-time":"2025-10-21T23:28:54Z","timestamp":1761089334000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719221000548"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,5]]},"references-count":19,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2021,5]]}},"alternative-id":["S1096719221000548"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2021.02.006","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2021,5]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Long-term impact of early initiation of enzyme replacement therapy in 34 MPS VI patients: A resurvey study","name":"articletitle","label":"Article Title"},{"value":"Molecular Genetics and Metabolism","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymgme.2021.02.006","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2021 The Authors. Published by Elsevier Inc.","name":"copyright","label":"Copyright"}]}}