Abstract
The development of enzyme replacement therapy for Gaucher disease was a triumph of translational medicine. What were the key steps in its development? What are the controversies surrounding its use?
MeSH terms
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Clinical Trials as Topic
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Dose-Response Relationship, Drug
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Gaucher Disease / complications
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Gaucher Disease / drug therapy*
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Gaucher Disease / physiopathology*
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Glucosylceramidase / administration & dosage
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Glucosylceramidase / therapeutic use*
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Humans
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Lectins, C-Type
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Mannose Receptor
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Mannose-Binding Lectins
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Patient Selection
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Receptors, Cell Surface
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Treatment Outcome
Substances
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Lectins, C-Type
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Mannose Receptor
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Mannose-Binding Lectins
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Receptors, Cell Surface
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Glucosylceramidase