leukoencephalopathy with vanishing white matter

ORPHA: 135

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 leukoencephalopathy with vanishing white matter, sourced from HPO and Orphanet clinical annotations.

Dysmyelinating leukodystrophyBrain imaging abnormalityOptic atrophyIrritabilitySeizureSpasticityCerebellar atrophyHyperreflexiaCerebral atrophyLimb ataxiaTruncal ataxiaProgressive neurologic deteriorationCerebellar vermis atrophyAtrophy/Degeneration affecting the brainstemPremature ovarian insufficiencyRenal hypoplasiaGonadal dysgenesisMicrocephalyCataractBlindnessAtypical behaviorApathyPrimary amenorrheaSecondary amenorrheaDysarthriaSpastic diplegiaHemiparesisMotor delayEncephalopathyDysmetriaFlexion contractureHepatosplenomegalyGrowth delayIntrauterine growth retardationDecreased fetal movementOligohydramniosPancreatitisVomitingDysphagiaMigraineHeadacheArthrogryposis multiplex congenitaLateral ventricle dilatationAbnormal pons morphologyNonketotic hyperglycinemiaFloppy infantMild global developmental delayFeeding difficultiesT2 hypointense thalamusWidened subarachnoid spaceDysgyriaGlobus pallidus hypointensity on susceptibility-weighted imagingCognitive impairmentOptic neuritisProgressive macrocephaly

Classification & Codes

Orphanet Code

ORPHA:135
leukoencephalopathy with vanishing white matter
OrphanetORPHA:135
Treatments0 drug(s)
Symptoms on record55 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
leukoencephalopathy with vanishing white matter | OrphanDrug