Mucopolysaccharidosis type II: Enzyme Replacement Therapy Efficiency
نویسندگان
چکیده
منابع مشابه
DRUG EVALUATION Idursulfase for enzyme-replacement therapy in mucopolysaccharidosis II
10.2217/14750708.4.3.231 © 2 Hunter syndrome (mucopolysaccharidosis II) is a rare, X-linked disorder caused by the missing or deficient lysosomal enzyme, iduronate-2-sulfatase, which leads to tissue and organ accumulation of glycosaminoglycans, resulting in multisystem dysfunction with death occurring most commonly in the first or second decade of life. Enzyme-replacement therapy with idursulfa...
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Recombinant mouse beta-glucuronidase administered intravenously to newborn mice with mucopolysaccharidosis type VII (MPS VII) is rapidly cleared from the circulation and localized in many tissues. Here we determine the tissue distribution of injected enzyme and describe its effects on the histopathology in 6-wk-old MPS VII mice that received either one injection of 28,000 U recombinant beta-glu...
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Hunter syndrome (or mucopolysaccharidosis type II [MPS II]) arises because of a deficiency in the lysosomal enzyme iduronate-2-sulfatase. Short stature is a prominent and consistent feature in MPS II. Enzyme replacement therapy (ERT) with idursulfase (Elaprase®) or idursulfase beta (Hunterase®) have been developed for these patients. The effect of ERT on the growth of Korean patients with Hunte...
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Antibodies against recombinant proteins can significantly reduce their effectiveness in unanticipated ways. We evaluated the humoral response of mice with the lysosomal storage disease mucopolysaccharidosis type I treated with weekly intravenous recombinant human alpha-l-iduronidase (rhIDU). Unlike patients, the majority of whom develop antibodies to recombinant human alpha-l-iduronidase, only ...
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We report evidence of a dose responsive effect of enzyme replacement therapy in mucopolysaccharidosis type VI cats from birth, at the clinical, biochemical, and histopathological level. Cats treated with weekly, intravenous recombinant human N-acetylgalactosamine-4-sulfatase at 1 and 5 mg/kg, were heavier, more flexible, had greatly reduced or no spinal cord compression, and had almost normal u...
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ژورنال
عنوان ژورنال: Current Pediatrics
سال: 2020
ISSN: 1682-5535,1682-5527
DOI: 10.15690/vsp.v18i5.2070