Direct Comparison of Measures of Endurance, Mobility, and Joint Function During Enzyme-Replacement T...
Direct Comparison of Measures of Endurance, Mobility, and Joint Function During Enzyme-Replacement Therapy of Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): Results After 48 Weeks in a Phase 2 Open-Label Clinical Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase
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United States: Am Acad Pediatrics
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Language
English
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Publisher
United States: Am Acad Pediatrics
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Objective. Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome) is a lysosomal storage disease caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase (ASB). This enzyme deficiency leads to a progressive disorder with multiple tissue and organ involvement. The disease is rare and is heterogeneous in its clinical presentation an...
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Full title
Direct Comparison of Measures of Endurance, Mobility, and Joint Function During Enzyme-Replacement Therapy of Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome): Results After 48 Weeks in a Phase 2 Open-Label Clinical Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase
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TN_cdi_proquest_miscellaneous_67893369
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_67893369
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ISSN
0031-4005
E-ISSN
1098-4275
DOI
10.1542/peds.2004-1023
