{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,12]],"date-time":"2026-09-12T02:13:43Z","timestamp":1789179223725,"version":"build-2803163510"},"reference-count":30,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2016,11,1]],"date-time":"2016-11-01T00:00:00Z","timestamp":1477958400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,9,5]],"date-time":"2016-09-05T00:00:00Z","timestamp":1473033600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.nationalarchives.gov.uk\/doc\/open-government-licence\/version\/3\/"}],"funder":[{"DOI":"10.13039\/100013995","name":"Sanofi Genzyme","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013995","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.jp","clinicalkey.com","clinicalkey.es","clinicalkey.com.au","clinicalkey.fr","jpeds.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["The Journal of Pediatrics"],"published-print":{"date-parts":[[2016,11]]},"DOI":"10.1016\/j.jpeds.2016.08.033","type":"journal-article","created":{"date-parts":[[2016,10,24]],"date-time":"2016-10-24T10:31:07Z","timestamp":1477305067000},"page":"219-226.e1","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":51,"special_numbering":"C","title":["Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I"],"prefix":"10.1016","volume":"178","author":[{"given":"Sarah","family":"Laraway","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean","family":"Mercer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elisabeth","family":"Jameson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jane","family":"Ashworth","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pauline","family":"Hensman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon A.","family":"Jones","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jpeds.2016.08.033_bib0010","series-title":"The online metabolic and molecular bases of inherited disease","first-page":"3421","article-title":"The mucopolysaccharidoses","author":"Neufeld","year":"2001"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0015","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1542\/peds.2008-0416","article-title":"Mucopolysaccharidosis I: management and treatment guidelines","volume":"123","author":"Muenzer","year":"2009","journal-title":"Pediatrics"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0020","doi-asserted-by":"crossref","first-page":"759","DOI":"10.1038\/gim.2014.25","article-title":"The natural history of MPS I: global perspectives from the MPS I Registry","volume":"16","author":"Beck","year":"2014","journal-title":"Genet Med"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0025","doi-asserted-by":"crossref","first-page":"581","DOI":"10.1016\/j.jpeds.2004.01.046","article-title":"Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase)","volume":"144","author":"Wraith","year":"2004","journal-title":"J Pediatr"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0030","doi-asserted-by":"crossref","first-page":"1320","DOI":"10.3324\/haematol.2011.058644","article-title":"Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy","volume":"97","author":"Saif","year":"2012","journal-title":"Haematologica"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0035","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/j.ymgme.2006.08.007","article-title":"A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years","volume":"90","author":"Sifuentes","year":"2007","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0040","doi-asserted-by":"crossref","first-page":"461","DOI":"10.1016\/j.pediatrneurol.2012.08.010","article-title":"Enzyme replacement therapy in an attenuated case of mucopolysaccharidosis type I (Scheie syndrome): a 6.5-year detailed follow-up","volume":"47","author":"Jurecka","year":"2012","journal-title":"Pediatr Neurol"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0045","doi-asserted-by":"crossref","first-page":"1259","DOI":"10.1093\/glycob\/cwq088","article-title":"Effect of 6 years of enzyme replacement therapy on plasma and urine glycosaminoglycans in attenuated MPS I patients","volume":"20","author":"Coppa","year":"2010","journal-title":"Glycobiology"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0050","doi-asserted-by":"crossref","first-page":"872","DOI":"10.1111\/j.1651-2227.2005.tb02004.x","article-title":"Clinical presentation and follow-up of patients with the attenuated phenotype of mucopolysaccharidosis type I","volume":"94","author":"Vijay","year":"2005","journal-title":"Acta Paediatr"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0055","doi-asserted-by":"crossref","first-page":"911","DOI":"10.1007\/s00431-011-1644-x","article-title":"Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry","volume":"171","author":"D'Aco","year":"2012","journal-title":"Eur J Pediatr"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0060","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1111\/j.1365-2214.2008.00918.x","article-title":"Psychosocial outcomes of bone marrow transplant for individuals affected by Mucopolysaccharidosis I Hurler Disease: patient social competency","volume":"35","author":"Pitt","year":"2009","journal-title":"Child Care Health Dev"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0065","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1542\/peds.2007-3847","article-title":"Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I","volume":"123","author":"Clarke","year":"2009","journal-title":"Pediatrics"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0070","doi-asserted-by":"crossref","first-page":"553","DOI":"10.1038\/sj.eye.6701921","article-title":"The ocular features of the mucopolysaccharidoses","volume":"20","author":"Ashworth","year":"2006","journal-title":"Eye (Lond)"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0075","doi-asserted-by":"crossref","first-page":"1353","DOI":"10.1001\/archopht.125.10.1353","article-title":"Ocular changes in patients with mucopolysaccharidosis I receiving enzyme replacement therapy: a 4-year experience","volume":"125","author":"Pitz","year":"2007","journal-title":"Arch Ophthalmol"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0080","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1056\/NEJM200101183440304","article-title":"Enzyme-replacement therapy in mucopolysaccharidosis I","volume":"344","author":"Kakkis","year":"2001","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0085","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1007\/s10545-007-0586-y","article-title":"Cardiac abnormalities in adults with the attenuated form of mucopolysaccharidosis type I","volume":"30","author":"Soliman","year":"2007","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0090","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.jpeds.2016.08.033_bib0095","doi-asserted-by":"crossref","first-page":"1341","DOI":"10.1016\/j.clinbiochem.2014.06.012","article-title":"Urinary glycosaminoglycan (uGAG) excretion in healthy pediatric and adolescent population","volume":"47","author":"Komosinska-Vassev","year":"2014","journal-title":"Clin Biochem"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0100","doi-asserted-by":"crossref","first-page":"1391","DOI":"10.1183\/09031936.00164109","article-title":"Global Lungs Initiative. Changes in the FEV\/FVC ratio during childhood and adolescence: an intercontinental study","volume":"36","author":"Quanjer","year":"2010","journal-title":"Eur Respir J"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0105","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1016\/j.ymgme.2014.10.012","article-title":"Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy","volume":"114","author":"Langereis","year":"2015","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0110","doi-asserted-by":"crossref","first-page":"e37","DOI":"10.1542\/peds.2006-2156","article-title":"Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase)","volume":"120","author":"Wraith","year":"2007","journal-title":"Pediatrics"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0115","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1007\/s10545-010-9059-9","article-title":"Efficacy of recombinant human alpha-L-iduronidase (laronidase) on restricted range of motion of upper extremities in mucopolysaccharidosis type I patients","volume":"33","author":"Tylki-Szymanska","year":"2010","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0120","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1007\/s10545-007-0551-9","article-title":"Enzyme replacement therapy in 12 patients with MPS I-H\/S with homozygous p.Leu490Pro mutation","volume":"30","author":"Arora","year":"2007","journal-title":"J Inherit Metab Dis"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0125","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1016\/j.ymgme.2013.04.023","article-title":"Does early use of enzyme replacement therapy alter the natural history of mucopolysaccharidosis I? Experience in three siblings","volume":"109","author":"Laraway","year":"2013","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0130","doi-asserted-by":"crossref","first-page":"e183","DOI":"10.1542\/peds.2009-1728","article-title":"Enzyme-replacement therapy in a 5-month-old boy with attenuated presymptomatic MPS I: 5-year follow-up","volume":"125","author":"Gabrielli","year":"2010","journal-title":"Pediatrics"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0135","doi-asserted-by":"crossref","first-page":"489","DOI":"10.1517\/14656566.6.3.489","article-title":"The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I","volume":"6","author":"Wraith","year":"2005","journal-title":"Expert Opin Pharmacother"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0140","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1016\/j.ymgme.2009.08.008","article-title":"Anthropometric data of 14 patients with mucopolysaccharidosis I:retrospective analysis and efficacy of recombinant human alpha-L-iduronidase (laronidase)","volume":"99","author":"Tylki-Szymanska","year":"2010","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0145","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1016\/j.ymgme.2013.11.015","article-title":"Early initiation of enzyme replacement therapy for the mucopolysaccharidoses","volume":"111","author":"Muenzer","year":"2014","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0150","doi-asserted-by":"crossref","first-page":"2164","DOI":"10.1182\/blood-2014-11-608075","article-title":"Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study","volume":"125","author":"Aldenhoven","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.jpeds.2016.08.033_bib0155","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1186\/s13023-015-0344-4","article-title":"Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships","volume":"10","author":"Al-Sannaa","year":"2015","journal-title":"Orphanet J Rare Dis"}],"container-title":["The Journal of Pediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0022347616307004?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0022347616307004?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2021,4,4]],"date-time":"2021-04-04T21:05:04Z","timestamp":1617570304000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0022347616307004"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,11]]},"references-count":30,"alternative-id":["S0022347616307004"],"URL":"https:\/\/doi.org\/10.1016\/j.jpeds.2016.08.033","relation":{},"ISSN":["0022-3476"],"issn-type":[{"value":"0022-3476","type":"print"}],"subject":[],"published":{"date-parts":[[2016,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I","name":"articletitle","label":"Article Title"},{"value":"The Journal of Pediatrics","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jpeds.2016.08.033","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Crown Copyright \u00a9 2016 Published by Elsevier Inc.","name":"copyright","label":"Copyright"}]}}