Treatment of Fabry Disease: Established and Emerging Therapies.
Ontology highlight
ABSTRACT:
SUBMITTER: Umer M
PROVIDER: S-EPMC9967779 | biostudies-literature | 2023 Feb
REPOSITORIES: biostudies-literature
Publications
Treatment of Fabry Disease: Established and Emerging Therapies.
Umer Muhammad M Kalra Dinesh K DK
Pharmaceuticals (Basel, Switzerland) 20230220 2
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 cerebrovascular systems. Early diagnosis and treatment are critical to prevent progression to irreversible tissue damage and organ failure, and to halt life-threatening complications that can significantly redu ...[more]
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