Fabry disease

GPTKB entity

Statements (31)
Predicate Object
gptkbp:instanceOf gptkb:genetic_disorder
gptkb:lysosomal_storage_disease
gptkbp:affects gptkb:skin
gptkb:nervous_system
heart
kidneys
alpha-galactosidase A enzyme
gptkbp:cause accumulation of globotriaosylceramide
gptkbp:causedBy mutation in GLA gene
gptkbp:diagnosedBy genetic testing
enzyme assay
gptkbp:firstDescribed 1898
gptkbp:inheritsFrom X-linked recessive
gptkbp:MeSH_ID D005176
gptkbp:namedAfter Johannes Fabry
gptkbp:OMIM 301500
gptkbp:prevalence rare
gptkbp:symptom gptkb:cardiomyopathy
renal failure
stroke
corneal verticillata
acroparesthesia
angiokeratoma
hypohidrosis
gptkbp:treatment gptkb:enzyme_replacement_therapy
chaperone therapy
gptkbp:bfsParent gptkb:Fabrazyme
gptkb:ST-920_(gene_therapy_for_Fabry_disease)
gptkb:Sangamo_Therapeutics
gptkbp:bfsLayer 7
https://www.w3.org/2000/01/rdf-schema#label Fabry disease

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