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Enzyme replacement therapy in Fabry disease
Journal of Inherited Metabolic Disease, 2001AbstractRecent clinical trials have demonstrated that enzyme replacement therapy with α‐galactosidase A (α‐Gal A) constitutes a major clinical advance in the treatment of patients with Fabry disease. This new therapeutic approach has been shown to be well tolerated and effective in reducing levels of the storage product globo‐triaosylceramide and in ...
R O, Brady +3 more
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Enzyme Replacement Therapy for Pompe Disease
Current Neurology and Neuroscience Reports, 2011Late-onset glycogenosis type II (glycogen storage disease type II [GSDII]) is a rare autosomal disorder caused by deficiency of acid maltase, a lysosomal enzyme that hydrolyzes glycogen to glucose. Recently, both infantile and adult GSDII patients have been treated with enzyme replacement therapy (ERT), and a number of studies including large cohorts ...
ANGELINI, CORRADO, SEMPLICINI, CLAUDIO
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Enzyme replacement therapy for Gaucher disease
Expert Opinion on Biological Therapy, 2008Gaucher disease is the most common lysosomal storage disease, and the first lysosomal storage disease for which a specific therapy has been developed. Enzyme replacement therapy, with glucocerebrosidase purified from human placentae, was introduced in 1991. Recombinant human glucocerebrosidase, produced by Chinese hamster ovary cells in tissue culture,
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2004
AbstractThis chapter traces the development of the field of enzyme replacement. It explores the major remaining problem — i.e., treatment of lysosomal storage diseases with a major neurologic component, because of insulation of the brain from the therapeutic enzyme by the blood-brain barrier.
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AbstractThis chapter traces the development of the field of enzyme replacement. It explores the major remaining problem — i.e., treatment of lysosomal storage diseases with a major neurologic component, because of insulation of the brain from the therapeutic enzyme by the blood-brain barrier.
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Enzyme replacement therapy for the management of the mucopolysaccharidoses
Int. Journal of Clinical Pharmacology and Therapeutics, 2009Enzyme replacement therapy (ERT) is now available for several of the mucopolysaccharidosis disorders. This brief review summarizes the role of ERT in reducing the burden of peripheral disease in many patients with mucopolysaccharidosis disorders, and describes the challenges that remain in treating the neurological manifestations of these conditions.
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Enzyme Replacement Therapy in Feline Mucopolysaccharidosis I
Molecular Genetics and Metabolism, 2001Enzyme replacement therapy (ERT) has long been considered an approach to treating lysosomal storage disorders caused by deficiency of lysosomal enzymes. ERT is currently used to treat Gaucher disease and is being developed for several lysosomal storage disorders now that recombinant sources of the enzymes have become available.
E D, Kakkis +11 more
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Pancreatic enzyme replacement therapy in chronic pancreatitis
Best Practice & Research Clinical Gastroenterology, 2010Exocrine pancreatic insufficiency (EPI) is a serious condition which occurs in several diseases including chronic pancreatitis (CP), cystic fibrosis, pancreatic cancer, and as a result of pancreatic surgery. The lack or absence of pancreatic enzymes leads to an inadequate absorption of fat, proteins, and carbohydrates, causing steatorrhoea and ...
Sikkens, Edmee +3 more
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Enzyme replacement therapy in a mouse model of aspartylglycosaminuria
The FASEB Journal, 2000ABSTRACT Aspartylglycosaminuria (AGU), the most common lysosomal disorder of glycoprotein degradation, is caused by deficient activity of glycosylasparaginase (AGA). AGA‐deficient mice share most of the clinical, biochemical and histopathologic characteristics of human AGU disease.
U, Dunder +7 more
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Enzyme replacement therapy for Anderson-Fabry disease
2010Anderson-Fabry disease is an X-linked defect of glycosphingolipid metabolism. Progressive renal insufficiency is a major source of morbidity, additional complications result from cardio- and cerebro-vascular involvement. Survival is reduced among affected males and symptomatic female carriers.To evaluate the effectiveness and safety of enzyme ...
Regina P, El Dib, Gregory M, Pastores
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