Erratum to: Enzyme replacement therapy for mucopolysaccharidosis VI—experience in Taiwan
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...
متن کاملEnzyme replacement therapy for murine mucopolysaccharidosis type VII.
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...
متن کاملOcular lesions in canine mucopolysaccharidosis I and response to enzyme replacement therapy.
PURPOSE Mucopolysaccharidosis I (MPS I) is an inherited metabolic disorder resulting from deficiency of α-L-iduronidase and lysosomal accumulation of glycosaminoglycans (GAG) in multiple tissues. Accumulation of GAG in corneal stromal cells causes corneal opacity and reduced vision. The purpose of this study was to determine the extent of ocular GAG accumulation and investigate the effectivenes...
متن کاملIntrathecal enzyme replacement therapy to treat spinal cord compression in mucopolysaccharidosis: Overview and rationale.
Spinal cord compression has been described in mucopolysaccharidosis I, II, IV, VI, and VII. The spinal cord compression in the mucopolysaccharidoses is believed to originate from narrowing of the vertebral canal due to bony abnormalities, and thickening of the meninges and spinal ligaments due to glycosaminoglycan infiltration [12]. The current standard of care involves surgical decompression. ...
متن کاملEnzyme replacement therapy for mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): experience in Hong Kong.
Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) is a very rare inherited lysosomal storage disease. We evaluated the efficacy and safety of weekly infusions of recombinant human arylsulfatase B as enzyme replacement therapy for two patients in whom this condition was advanced. The primary outcome variables were the distance walked in a 6-minute walk test, forced vital capacity, and ejection ...
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ژورنال
عنوان ژورنال: Journal of Inherited Metabolic Disease
سال: 2010
ISSN: 0141-8955,1573-2665
DOI: 10.1007/s10545-010-9233-0