Fabry disease

MeSH: D000795ORPHA: 324

Overview

rare human genetic lysosomal storage disorder

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Clinical Presentation

Signs and symptoms associated with Fabry disease, sourced from HPO and Orphanet clinical annotations.

Renal insufficiencyNephrotic syndromeHearing impairmentConjunctival telangiectasiaHematuriaHyperkeratosisHypohidrosisAngiokeratomaCorneal dystrophyArthritisSubcutaneous noduleCongestive heart failureAnemiaMalabsorptionAbdominal painTransient ischemic attackArthralgiaMyalgiaAbnormal glycosphingolipid metabolismCorneal opacityFatigueElevated circulating globotriaosylceramide concentrationDecreased alpha-galactosidase A activityMucosal telangiectasiaeTelangiectasia of the skinAbnormal renal tubule morphologyProteinuriaNephropathyThick lower lip vermilionCoarse facial featuresTinnitusCataractOptic atrophyAtypical behaviorDelayed pubertyAbnormal aortic valve morphologyMitral regurgitationAtrioventricular blockNausea and vomitingAnorexiaHeat intoleranceEmphysemaHyperlipidemiaAbnormal circulating lipid concentrationExercise intoleranceShort statureBundle branch blockChronic painAcroparesthesiaCognitive impairmentCornea verticillataSensorineural hearing impairmentDepressionAnxietyHypertensionDiabetes insipidusLymphedemaSeizureStrokeAbnormal myocardium morphologyHypertrophic cardiomyopathyAngina pectorisLeft ventricular hypertrophyFeverRespiratory insufficiencyDyspneaVertigoDevelopmental regressionAchalasiaAbnormal femur morphologyAbnormal endocardium morphologyReduced bone mineral densityChronic pulmonary obstructionArrhythmiaGlomerulopathyHyperhidrosis

Classification & Codes

MeSH Code

D000795

Orphanet Code

ORPHA:324
Fabry disease
MeSHD000795
OrphanetORPHA:324
Treatments0 drug(s)
Symptoms on record76 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO