Fabry Disease D000795

Related MeSH Hierarchy (12)

Description

An X-linked inherited metabolic disease caused by a deficiency of lysosomal ALPHA-GALACTOSIDASE A. It is characterized by intralysosomal accumulation of globotriaosylceramide and other GLYCOSPHINGOLIPIDS in blood vessels throughout the body leading to multi-system complications including renal, cardiac, cerebrovascular, and skin disorders.   MeSH

Approved Indicated Drugs (1)

Phase 3 Indicated Drugs (2)

Phase 2 Indicated Drugs (2)


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