{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,12]],"date-time":"2026-10-12T11:52:32Z","timestamp":1791805952527,"version":"4.3.4"},"reference-count":23,"publisher":"Elsevier BV","issue":"5","license":[{"start":{"date-parts":[[2004,5,1]],"date-time":"2004-05-01T00:00:00Z","timestamp":1083369600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["The Journal of Pediatrics"],"published-print":{"date-parts":[[2004,5]]},"DOI":"10.1016\/j.jpeds.2004.03.018","type":"journal-article","created":{"date-parts":[[2004,5,12]],"date-time":"2004-05-12T10:26:10Z","timestamp":1084357570000},"page":"574-580","source":"Crossref","is-referenced-by-count":231,"title":["Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)"],"prefix":"10.1016","volume":"144","author":[{"given":"Paul","family":"Harmatz","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chester B","family":"Whitley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lewis","family":"Waber","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ray","family":"Pais","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Steiner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Plecko","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paige","family":"Kaplan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julie","family":"Simon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ellen","family":"Butensky","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John J","family":"Hopwood","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jpeds.2004.03.018_BIB1","series-title":"The metabolic basis of inherited disease","first-page":"2465","article-title":"The mucopolysaccharidoses","author":"Neufeld","year":"1995"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB2","doi-asserted-by":"crossref","first-page":"389","DOI":"10.1007\/BF00206770","article-title":"An update on the frequency of mucopolysaccharide syndromes in British Columbia","volume":"85","author":"Lowry","year":"1990","journal-title":"Hum Genet"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB3","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1001\/jama.281.3.249","article-title":"Prevalence of lysosomal storage disorders","volume":"281","author":"Meikle","year":"1999","journal-title":"JAMA"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB4","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1007\/s004390050641","article-title":"Incidence of the mucopolysaccharidoses in Northern Ireland","volume":"101","author":"Nelson","year":"1997","journal-title":"Hum Genet"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB5","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1007\/s004399900075","article-title":"The frequency of lysosomal storage diseases in the Netherlands","volume":"105","author":"Poorthuis","year":"1999","journal-title":"Hum Genet"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB6","doi-asserted-by":"crossref","first-page":"1606","DOI":"10.1056\/NEJM198412203112504","article-title":"Bone-marrow transplantation in the Maroteaux-Lamy syndrome (mucopolysaccharidosis Type VI). Biochemical and clinical status 24 months after transplantation","volume":"311","author":"Krivit","year":"1984","journal-title":"N Engl J Med"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB7","first-page":"47","article-title":"Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). Treatment by allogeneic bone marrow transplantation in 6 patients and potential for autotransplantation bone marrow gene insertion","volume":"7","author":"Krivit","year":"1992","journal-title":"Int Pediatr"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB8","doi-asserted-by":"crossref","first-page":"1398","DOI":"10.1016\/S0140-6736(95)92597-X","article-title":"Allogeneic bone marrow transplantation for lysosomal storage diseases","volume":"246","author":"Hoogerbrugge","year":"1995","journal-title":"Lancet"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB9","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1017\/S0012162201001165","article-title":"Advances in the treatment of lysosomal storage disease","volume":"43","author":"Wraith","year":"2001","journal-title":"Dev Med Child Neurol"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB10","doi-asserted-by":"crossref","first-page":"36335","DOI":"10.1074\/jbc.274.51.36335","article-title":"Advantages of using same species enzyme for replacement therapy in a feline model of mucopolysaccharidosis type VI","volume":"274","author":"Bielicki","year":"1999","journal-title":"J Biol Chem"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB11","doi-asserted-by":"crossref","first-page":"1864","DOI":"10.1172\/JCI118617","article-title":"Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome","volume":"97","author":"Crawley","year":"1996","journal-title":"J Clin Invest"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB12","doi-asserted-by":"crossref","first-page":"374","DOI":"10.1093\/clinchem\/35.3.374","article-title":"Diagnostic test for mucopolysaccharidosis I. Direct method for quantifying excessive urinary glycosaminoglycan excretion","volume":"35","author":"Whitley","year":"1989","journal-title":"Clin Chem"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB13","doi-asserted-by":"crossref","first-page":"362","DOI":"10.1016\/S0022-3476(96)70067-7","article-title":"A six-minute walk test for assessing exercise tolerance in severely ill children","volume":"129","author":"Nixon","year":"1996","journal-title":"J Pediatr"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB14","doi-asserted-by":"crossref","first-page":"1761","DOI":"10.1002\/art.1780371209","article-title":"Measurement of health status in children with juvenile rheumatoid arthritis","volume":"37","author":"Singh","year":"1994","journal-title":"Arth Rheum"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB15","doi-asserted-by":"crossref","first-page":"152","DOI":"10.1111\/j.1745-7599.1991.tb01094.x","article-title":"A new developmental screening test. The Denver II","volume":"3","author":"Sperhac","year":"1991","journal-title":"J Am Acad Nurse Pract"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB16","unstructured":"Byers S, Auclair D, Brumfield LK, Robinson A, Hopwood JJ. Localised therapy for joint disease in MPS VI. 2002. Paris, 7th International Symposium on MPS and Related Diseases. 6-20-2002."},{"key":"10.1016\/j.jpeds.2004.03.018_BIB17","doi-asserted-by":"crossref","first-page":"1319","DOI":"10.1038\/labinvest.3780345","article-title":"Articular chondrocytes from animals with a dermatan sulfate storage disease undergo a high rate of apoptosis and release nitric oxide and inflammatory cytokines: a possible mechanism underlying degenerative joint disease in the mucopolysaccharidoses","volume":"81","author":"Simonaro","year":"2001","journal-title":"Lab Invest"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB18","doi-asserted-by":"crossref","first-page":"2081","DOI":"10.1182\/blood.V93.6.2081.406k07_2081_2088","article-title":"Immunosurveillance of alglucerase enzyme therapy for gaucher patients: induction of humoral tolerance in seroconverted patients after repeat administration","volume":"93","author":"Rosenberg","year":"1999","journal-title":"Blood"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB19","doi-asserted-by":"crossref","first-page":"651","DOI":"10.1172\/JCI119208","article-title":"Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI","volume":"99","author":"Crawley","year":"1997","journal-title":"J Clin Invest"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB20","first-page":"104","article-title":"Newborn screening for lysosomal storage disorders","volume":"30","author":"Meikle","year":"1999","journal-title":"Southeast Asian J Trop Med Public Health"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB21","doi-asserted-by":"crossref","first-page":"2074","DOI":"10.1093\/clinchem\/35.10.2074","article-title":"Diagnostic test for mucopolysaccharidosis II. Rapid quantification of glycosaminoglycan in urine samples collected on a paper matrix","volume":"35","author":"Whitley","year":"1989","journal-title":"Clin Chem"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB22","doi-asserted-by":"crossref","first-page":"56","DOI":"10.1006\/mgme.2001.3271","article-title":"Urinary glycosaminoglycan excretion quantified by an automated semimicro method in specimens conveniently transported from around the globe","volume":"75","author":"Whitley","year":"2002","journal-title":"Mol Genet Metabol"},{"key":"10.1016\/j.jpeds.2004.03.018_BIB23","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1023\/A:1010379320378","article-title":"Enzyme replacement therapy in mucopolysaccharidosis type I: progress and emerging difficulties","volume":"24","author":"Wraith","year":"2001","journal-title":"J Inherit Metab Dis"}],"container-title":["The Journal of Pediatrics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S002234760400215X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S002234760400215X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2020,4,2]],"date-time":"2020-04-02T03:58:16Z","timestamp":1585799896000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S002234760400215X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2004,5]]},"references-count":23,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2004,5]]}},"alternative-id":["S002234760400215X"],"URL":"https:\/\/doi.org\/10.1016\/j.jpeds.2004.03.018","relation":{},"ISSN":["0022-3476"],"issn-type":[{"value":"0022-3476","type":"print"}],"subject":[],"published":{"date-parts":[[2004,5]]}}}