PYCR2-related microcephaly-progressive leukoencephalopathy

ORPHA: 481152

Overview

human disease

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 PYCR2-related microcephaly-progressive leukoencephalopathy, sourced from HPO and Orphanet clinical annotations.

Progressive microcephalyIntellectual disabilityAbsent speechFailure to thriveInability to walkSevere global developmental delayFeeding difficultiesHypoplasia of the maxillaLow-set earsProtruding earBulbous noseSeizureHypotoniaSpasticityAbnormal facial shapeVomitingHypoplasia of the corpus callosumDevelopmental regressionSkeletal muscle atrophyCNS hypomyelinationSevere demyelination of the white matterHyperintensity of cerebral white matter on MRIHigh palateThin vermilion borderHypertelorismSmooth philtrumTriangular faceNarrow foreheadLong philtrumHearing impairmentOverfolded helixMacrotiaAnteverted naresDownslanted palpebral fissuresEsotropiaUpslanted palpebral fissureNystagmusAggressive behaviorIrritabilityPectus carinatumAbnormality of the skeletal systemArachnodactylyAtaxiaAgenesis of corpus callosumGait disturbanceHyperreflexiaFlexion contractureJoint hypermobilityBilateral tonic-clonic seizureGlobal brain atrophyHypoplasia of the brainstemPoor speechLimb hypertoniaHip dislocationWrist hypermobilityThoracic kyphoscoliosisAnkle hypermobilityBroad halluxFocal myoclonic seizureBroad eyebrowBroad thumbCerebral visual impairment

Classification & Codes

Orphanet Code

ORPHA:481152
PYCR2-related microcephaly-progressive leukoencephalopathy
OrphanetORPHA:481152
Treatments0 drug(s)
Symptoms on record62 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO