homocystinuria

MeSH: D006712ORPHA: 394

Overview

amino acid metabolic disorder that involves an accumulation of homocysteine in the serum and an increased excretion of homocysteine in the urine

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 homocystinuria, sourced from HPO and Orphanet clinical annotations.

Dental crowdingOsteoporosisEctopia lentisArachnodactylyIntellectual disabilityDisproportionate tall statureHyperhomocystinemiaRecurrent fracturesAbnormality of amino acid metabolismMyopiaAmblyopiaPectus excavatumPectus carinatumHypertensionJoint stiffnessPes cavusPulmonary embolismSparse scalp hairCerebral ischemiaScoliosisKyphosisGenu valgumArterial thrombosisVenous thrombosisArteriovenous malformationHigh palateStrabismusGlaucomaCataractRetinal detachmentOptic atrophyAtypical behaviorPsychosisDepressionAutistic behaviorAnxietyHypopigmentation of the skinUrticariaLens subluxationSeizureParkinsonismSpecific learning disabilityDystoniaPancreatitisSubcutaneous hemorrhageAnorexiaEsophageal varixAbnormality of extrapyramidal motor functionIntracranial hemorrhageGastrointestinal hemorrhageHepatomegalyEEG abnormalityAbnormal cerebral white matter morphologyElevated circulating hepatic transaminase concentrationHemiplegia/hemiparesisAbnormal retinal pigmentationObsessive-compulsive traitNeurodevelopmental delayMalar rashLivedo reticularisCerebral venous sinus thrombosisHernia

Classification & Codes

MeSH Code

D006712

Orphanet Code

ORPHA:394
homocystinuria
MeSHD006712
OrphanetORPHA:394
Treatments0 drug(s)
Symptoms on record62 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO