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https://hdl.handle.net/2440/7622
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Type: | Journal article |
Title: | Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome) |
Author: | Harmatz, P. Whitley, C. Waber, L. Pais, R. Steiner, R. Plecko, B. Kaplan, P. Simon, J. Butensky, E. Hopwood, J. |
Citation: | Journal of Pediatrics, 2004; 144(5):574-580 |
Publisher: | Mosby Inc |
Issue Date: | 2004 |
ISSN: | 0022-3476 1097-6833 |
Statement of Responsibility: | Paul Harmatz, Chester B Whitley, Lewis Waber, Ray Pais, Robert Steiner, Barbara Plecko, Paige Kaplan, Julie Simon, Ellen Butensky, John J Hopwood |
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. |
Keywords: | Humans Mucopolysaccharidosis VI N-Acetylgalactosamine-4-Sulfatase Recombinant Proteins Statistics, Nonparametric Double-Blind Method Dose-Response Relationship, Drug Adolescent Child Female Male |
DOI: | 10.1016/j.jpeds.2004.03.018 |
Published version: | http://dx.doi.org/10.1016/j.jpeds.2004.03.018 |
Appears in Collections: | Aurora harvest 4 Paediatrics publications |
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