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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
2016
Review
2016
Enzyme replacement therapy with laronidase (Aldurazyme(®)) for treating mucopolysaccharidosis type I.
BACKGROUND Mucopolysaccharidosis type I can be classified as three clinical sub-types; Hurler syndrome, Hurler-Scheie syndrome… 
2014
2014
The generation of induced pluripotent stem cells (iPSCs) from patients with infantile and late-onset types of Pompe disease and the effects of treatment with acid-α-glucosidase in Pompe's iPSCs.
Review
2012
Review
2012
Enzyme replacement therapy for lysosomal storage diseases.
Enzyme replacement therapy (ERT) has been approved for 6 lysosomal storage diseases (LSDs) worldwide including Japan. These… 
2010
2010
Enzyme replacement therapy and Fabry nephropathy.
Involvement of the kidneys in Fabry disease ("nephropathy") occurs in male and female individuals. The majority of patients with… 
Review
2010
Review
2010
Dialysis and transplantation in Fabry disease: indications for enzyme replacement therapy.
ESRD is a major cause of morbidity and premature mortality in Fabry disease, particularly in classically affected males. The… 
Review
2008
Review
2008
Update on Fabry disease: kidney involvement, renal progression and enzyme replacement therapy.
Fabry disease is an X-linked lysosomal storage disorder which is caused by a deficiency of the lysosomal enzyme alpha… 
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… 
2000
2000
Enzyme Replacement Therapy in a Feline Model of MPS VI: Modification of Enzyme Structure and Dose Frequency
Enzyme replacement therapy (ERT) in the MPS VI cat is effective at reducing or eliminating pathology in most connective tissues… 
Highly Cited
1994
Highly Cited
1994
Stoichiometry of recombinant cystic fibrosis transmembrane conductance regulator in epithelial cells and its functional reconstitution into cells in vitro.
1973
1973
Caveat to protein replacement therapy for genetic disease. Immunological implications of accurate molecular diagnosis.
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