Abstract
Fabry disease is a rare genetic lysosomal storage disorder characterized by a deficiency of the enzyme alpha-galactosidase A. The recent availability of enzyme-replacement therapy with agalsidase alfa offers specific treatment for this serious, progressive condition.
Publication types
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Research Support, Non-U.S. Gov't
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Review
MeSH terms
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Aged
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Drug Costs
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Fabry Disease / drug therapy*
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Fabry Disease / economics
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Fabry Disease / genetics
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Female
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Humans
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Isoenzymes / deficiency
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Isoenzymes / economics
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Isoenzymes / therapeutic use*
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Male
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Recombinant Proteins
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Treatment Outcome
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alpha-Galactosidase / economics
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alpha-Galactosidase / therapeutic use*
Substances
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Isoenzymes
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Recombinant Proteins
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agalsidase alfa
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alpha-Galactosidase