Enzyme Replacement Therapy

Known as: Enzyme Replacement Therapies, Replacement Therapies, Enzyme, Protein Replacement Therapy 
Therapeutic replacement or supplementation of defective or missing enzymes to alleviate the effects of enzyme deficiency (e.g., GLUCOSYLCERAMIDASE… (More)
National Institutes of Health

Papers overview

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Highly Cited
2010
Highly Cited
2010
Mucopolysaccharidosis type VI (MPS VI) is a progressive, multisystem disorder caused by a deficiency of the lysosomal enzyme N… (More)
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Highly Cited
2009
Highly Cited
2009
BACKGROUND Enzyme replacement therapy with recombinant alpha-galactosidase A reduces left ventricular hypertrophy and improves… (More)
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Highly Cited
2007
Highly Cited
2007
AIM To assess the effects of enzyme replacement therapy (ERT) in children with Fabry disease. METHODS Safety and efficacy of… (More)
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Highly Cited
2006
Highly Cited
2006
Enzyme replacement therapy (ERT) with recombinant human alpha-galactosidase A (r-halphaGalA) enhances microvascular… (More)
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Highly Cited
2006
Highly Cited
2006
In Pompe disease, a genetic deficiency of lysosomal acid α-glucosidase, glycogen accumulates abnormally in the lysosomes of… (More)
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Highly Cited
2005
Highly Cited
2005
Enzyme replacement therapy (ERT) effectively reverses storage in several lysosomal storage diseases. However, improvement in… (More)
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Highly Cited
2003
Highly Cited
2003
BACKGROUND Enzyme replacement therapy (ERT) has been shown to enhance microvascular endothelial globotriaosylceramide clearance… (More)
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Highly Cited
2003
Highly Cited
2003
Fabry disease is a lysosomal storage disease arising from deficiency of the enzyme alpha-galactosidase A. Two recombinant protein… (More)
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Highly Cited
2002
Highly Cited
2002
Hematopoietic stem cell (HSC) gene therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID) has… (More)
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Highly Cited
2002
Highly Cited
2002
PURPOSE Gaucher disease is the first lysosomal storage disorder to be treated with macrophage-targeted enzyme replacement therapy… (More)
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