Title |
Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long‐term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans
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Published in |
Journal of Inherited Metabolic Disease, September 2012
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DOI | 10.1007/s10545-012-9538-2 |
Pubmed ID | |
Authors |
Minke H. de Ru, Linda van der Tol, Naomi van Vlies, Brian W. Bigger, Carla E. M. Hollak, Lodewijk IJlst, Wim Kulik, Henk van Lenthe, Muhammad A. Saif, Tom Wagemans, Willem M. van der Wal, Ronald J. Wanders, Frits A. Wijburg |
Abstract |
Mucopolysaccharidosis type I (MPS I) results in a defective breakdown of the glycosaminoglycans (GAGs) heparan sulfate and dermatan sulfate, which leads to a progressive disease. Enzyme replacement therapy (ERT) results in clearance of these GAGs from a range of tissues and can significantly ameliorate several symptoms. The biochemical efficacy of ERT is generally assessed by the determination of the total urinary excretion of GAGs. However, this has limitations. We studied the concentrations of heparan sulfate and dermatan sulfate derived disaccharides (HS and DS, respectively) in the plasma and urine of seven patients and compared these levels with total urinary GAGs (uGAGs) levels. |
X Demographics
Geographical breakdown
Country | Count | As % |
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Mexico | 1 | 50% |
Unknown | 1 | 50% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 2 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Unknown | 44 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Ph. D. Student | 14 | 32% |
Researcher | 8 | 18% |
Student > Master | 4 | 9% |
Other | 3 | 7% |
Student > Bachelor | 2 | 5% |
Other | 4 | 9% |
Unknown | 9 | 20% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 12 | 27% |
Biochemistry, Genetics and Molecular Biology | 9 | 20% |
Agricultural and Biological Sciences | 6 | 14% |
Chemistry | 4 | 9% |
Nursing and Health Professions | 1 | 2% |
Other | 2 | 5% |
Unknown | 10 | 23% |