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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… 
National Institutes of Health

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3 relations
Enzyme Therapynursing therapystandards characteristics

Papers overview

Semantic Scholar uses AI to extract papers important to this topic.
Review
2014
Review
2014
Appropriateness of Newborn Screening for α1-Antitrypsin Deficiency
Objective: The Alpha-1 Foundation convened a workshop to consider the appropriateness of newborn screening for &agr;-1… 
Review
2010
Review
2010
Velaglucerase alfa, a human recombinant glucocerebrosidase enzyme replacement therapy for type 1 Gaucher disease.
Gaucher disease (GD) is caused by a deficiency of the lysosomal enzyme glucocerebrosidase, which results in the accumulation of… 
2009
2009
Advancements in gene transfer-based therapy for hemophilia A
Gene therapy has promised clinical benefit to those suffering with hemophilia A, but this benefit has not yet been realized… 
Review
2009
Review
2009
Enzyme replacement therapy for the management of the mucopolysaccharidoses.
Enzyme replacement therapy (ERT) is now available for several of the mucopolysaccharidosis disorders. This brief review… 
2007
2007
Elevated endothelial microparticles in Fabry children decreased after enzyme replacement therapy.
To the Editor: Fabry disease (FD), an X-linked metabolic disorder, is caused by insufficient activity of the lysosomal enzyme… 
2006
2006
Platelet function and coagulation abnormalities in type 1 Gaucher disease patients: effects of enzyme replacement therapy (ERT)
All individuals or their parents gave informed consent to the study. Laboratory tests were performed at initial diagnosis and, at… 
Review
2004
Review
2004
Onco-retroviral and lentiviral vector-based gene therapy for hemophilia: preclinical studies.
Hemophilia A and B gene therapy requires long-term and stable expression of coagulation factor VIII (FVIII) or factor IX (FIX… 
1998
1998
Enzyme replacement therapy decreases hypergammaglobulinemia in Gaucher's disease.
We report the effects of enzyme replacement therapy in a patient with Gaucher's disease associated with a monoclonal gammopathy… 
1997
1997
Bone marrow relaxation times in Gaucher disease before and after enzyme replacement therapy
Abstract. The aim of this work was to monitor the effectiveness of enzyme replacement therapy on the basis of the changes in T1… 
1996
1996
Regression of skeletal changes in type 1 Gaucher disease with enzyme replacement therapy
Abstract A 9-year-old patient presented with an acute onset of pain of the right femur showing destructive changes with… 
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