Please use this identifier to cite or link to this item: https://hdl.handle.net/2440/7622
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dc.contributor.authorHarmatz, P.-
dc.contributor.authorWhitley, C.-
dc.contributor.authorWaber, L.-
dc.contributor.authorPais, R.-
dc.contributor.authorSteiner, R.-
dc.contributor.authorPlecko, B.-
dc.contributor.authorKaplan, P.-
dc.contributor.authorSimon, J.-
dc.contributor.authorButensky, E.-
dc.contributor.authorHopwood, J.-
dc.date.issued2004-
dc.identifier.citationJournal of Pediatrics, 2004; 144(5):574-580-
dc.identifier.issn0022-3476-
dc.identifier.issn1097-6833-
dc.identifier.urihttp://hdl.handle.net/2440/7622-
dc.description.abstract<h4>Objectives</h4>To evaluate the safety and efficacy of weekly treatment with human recombinant N-acetylgalactosamine 4-sulfatase (rhASB) in humans with mucopolysaccharidosis type VI (MPS VI).<h4>Study design</h4>An ongoing Phase I/II, randomized, two-dose, double-blind study. Patients were randomized to weekly infusions of either high (1.0 mg/kg) or low (0.2 mg/kg) doses of rhASB. Six patients (3 male, 3 female; age 7-16 years) completed at least 24 weeks of treatment, five of this group have completed at least 48 weeks.<h4>Results</h4>No drug-related serious adverse events, significant laboratory abnormalities, or allergic reactions were observed in the study. The high-dose group experienced a more rapid and larger relative reduction in urinary glycosaminoglycan that was sustained through week 48. Improvements in the 6-minute walk test were observed in all patients with dramatic gains in those walking <100 meters at baseline. Shoulder range of motion improved in all patients at week 48 and joint pain improved in patients with significant pain at baseline.<h4>Conclusions</h4>rhASB treatment was well-tolerated and reduced lysosomal storage as evidenced by a dose-dependent reduction in urinary glycosaminoglycan. Clinical responses were present in all patients, but the largest gains occurred in patients with advanced disease receiving high-dose rhASB.-
dc.description.statementofresponsibilityPaul Harmatz, Chester B Whitley, Lewis Waber, Ray Pais, Robert Steiner, Barbara Plecko, Paige Kaplan, Julie Simon, Ellen Butensky, John J Hopwood-
dc.language.isoen-
dc.publisherMosby Inc-
dc.source.urihttp://dx.doi.org/10.1016/j.jpeds.2004.03.018-
dc.subjectHumans-
dc.subjectMucopolysaccharidosis VI-
dc.subjectN-Acetylgalactosamine-4-Sulfatase-
dc.subjectRecombinant Proteins-
dc.subjectStatistics, Nonparametric-
dc.subjectDouble-Blind Method-
dc.subjectDose-Response Relationship, Drug-
dc.subjectAdolescent-
dc.subjectChild-
dc.subjectFemale-
dc.subjectMale-
dc.titleEnzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome)-
dc.typeJournal article-
dc.identifier.doi10.1016/j.jpeds.2004.03.018-
pubs.publication-statusPublished-
Appears in Collections:Aurora harvest 4
Paediatrics publications

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