Treatment of Fabry Disease: Established and Emerging Therapies
Fabry disease (FD) is a rare, X-linked inherited disorder of glycosphingolipid metabolism. It leads to the progressive accumulation of globotriaosylceramide within lysosomes due to a deficiency of α-galactosidase A enzyme. It involves multiple organs, predominantly the renal, cardiac, and cerebrovas...
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| Main Authors: | , |
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| Format: | Book |
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MDPI AG,
2023-02-01T00:00:00Z.
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| Call Number: |
A1234.567 |
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