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Fabry disease

ORPHA:324· ICD-10 E75.2

Definition

A rare genetic, multisystemic lysosomal disease characterized by specific cutaneous (angiokeratoma), neurological (pain), renal (proteinuria, chronic kidney failure), cardiovascular (cardiomyopathy, arrhythmia), cochleo-vestibular and cerebrovascular manifestations (transient ischemic attacks, strokes). The phenotypic expression depends on age of onset and, in females, the level of X-inactivation.

Prevalence
1-9 / 1 000 000
Inheritance
X-linked dominant, X-linked recessive
Age of onset
Adolescent, Adult, Childhood