Aicardi-Goutieres syndrome

MeSH: C535607ORPHA: 51

Overview

a rare genetic neurodevelopmental 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 Aicardi-Goutieres syndrome, sourced from HPO and Orphanet clinical annotations.

Convex nasal ridgeGait disturbanceSpasticityGlobal developmental delayHypertoniaPorencephalic cystArrhinencephalyProfound intellectual disabilityMultifocal cerebral white matter abnormalitiesMicrocephalyEyelid colobomaIrritabilityDry skinSeizureDystoniaHepatosplenomegalyUnexplained feversAbnormality of extrapyramidal motor functionHypoplasia of the corpus callosumVentriculomegalyLoss of speechDevelopmental regressionLeukodystrophyCerebral calcificationElevated circulating hepatic transaminase concentrationAutoimmunityShort statureHemiplegia/hemiparesisExtrapyramidal muscular rigidityAxial hypotoniaChronic CSF lymphocytosisIncreased CSF interferon alphaChilblainsBrain atrophyIncreased circulating interferon-gamma concentrationMicropenisLow-set earsAbnormality of eye movementGlaucomaPtosisNystagmusDiabetes mellitusHypothyroidismCutis marmorataAcrocyanosisDevelopmental glaucomaTremorPlagiocephalyArthritisHoarse voiceCardiomegalySpastic paraparesisHeadacheSpastic tetraplegiaScoliosisMultiple joint contracturesMuscle stiffnessNeonatal alloimmune thrombocytopeniaProlonged neonatal jaundiceDemyelinating peripheral neuropathyAbnormal pyramidal signPanniculitisRaynaud phenomenonHypertrophic cardiomyopathyAortic aneurysmCalcification of the aortaChronic lymphatic leukemiaMoyamoya phenomenonEnchondromaDegeneration of the striatumLipoatrophyMyositis

Classification & Codes

MeSH Code

C535607

Orphanet Code

ORPHA:51
Aicardi-Goutieres syndrome
MeSHC535607
OrphanetORPHA:51
Treatments0 drug(s)
Symptoms on record72 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO