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Idursulfase in Hunter syndrome treatment
Drugs of Today, 2007Hunter syndrome (mucopolysaccharidosis II, MPS II) is a rare X-linked lysosomal storage disorder caused by the deficiency of enzyme iduronate-2-sulfatase (I2S), which results in accumulation of undegraded dermatan and heparan sulfate in various tissues and organs. Enzyme replacement therapy with Elaprase (idursulfase, a recently approved orphan product)
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Idursulfase for the treatment of mucopolysaccharidosis II
Expert Opinion on Pharmacotherapy, 2008Human recombinant proteins are being used to treat an increasing number of disorders. Advances in the large scale production of recombinant proteins and the understanding of glycosylation and its importance for protein targeting and function have led to the development of recombinant enzyme-replacement regimens for a number of human lysosomal storage ...
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Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II
Acta Paediatrica, 2008AbstractMucopolysaccharidosis type II (MPS II; Hunter syndrome) is a rare X‐linked recessive disease caused by deficiency of the lysosomal enzyme iduronate‐2‐sulphatase. This leads to the progressive accumulation of glycosaminoglycans in tissues throughout the body.
Wraith, James, Wraith, J. Edmond
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Idursulfase: enzyme replacement therapy for mucopolysaccharidosis Type II (Hunter syndrome)
Expert Review of Endocrinology & Metabolism, 2007Mucopolysaccharidosis type II (Hunter syndrome) is a lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase leading to tissue accumulation of glycosaminoglycans. It manifests with short stature, joint stiffness, coarse facial features, hepatosplenomegaly, and progressive mental retardation.
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Idursulfase for enzyme-replacement therapy in mucopolysaccharidosis II
Therapy, 2007Hunter 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.
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Hypersensitivity Reaction and a Single‐Bag Rapid Desensitization to Idursulfase
American Journal of Medical Genetics Part AABSTRACTIdursulfase is the first‐line and only available enzyme replacement therapy (ERT) for Mucopolysaccharidosis type II (MPS II), or Hunter Syndrome. Deficiency in the lysosomal enzyme iduronate‐2‐sulfatase leads to progressive skeletal deformities, neurologic deterioration, airway obstruction, and cardiomyopathy. In severe cases, these deformities
Jenna H. Jung +3 more
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