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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… Expand
National Institutes of Health

Papers overview

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Highly Cited
2015
Highly Cited
2015
Your love and endless support keep me afloat. iv ACKNOWLEDGEMENTS I would like to acknowledge those whose guidance and support… Expand
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Highly Cited
2010
Highly Cited
2010
McGill JJ, Inwood AC, Coman DJ, Lipke ML, de Lore D, Swiedler SJ, Hopwood JJ. Enzyme replacement therapy for… Expand
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Highly Cited
2009
Highly Cited
2009
Background— Enzyme replacement therapy with recombinant α-galactosidase A reduces left ventricular hypertrophy and improves… Expand
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Highly Cited
2009
Highly Cited
2009
Late-onset glycogen storage disease type 2 (GSD2)/Pompe disease is a progressive multi-system disease evoked by a deficiency of… Expand
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Highly Cited
2008
Highly Cited
2008
OBJECTIVE. The goal was to analyze the clinical responses to enzyme replacement therapy with alglucerase or imiglucerase in a… Expand
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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… Expand
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Highly Cited
2003
Highly Cited
2003
Background—Enzyme replacement therapy (ERT) has been shown to enhance microvascular endothelial globotriaosylceramide clearance… Expand
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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… Expand
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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… Expand
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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… Expand
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