metachromatic leukodystrophy

MeSH: D007966ORPHA: 512

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 metachromatic leukodystrophy, sourced from HPO and Orphanet clinical annotations.

Periventricular leukomalaciaAbnormal circulating enzyme concentration or activityHearing impairmentVisual impairmentAbnormality of visual evoked potentialsDecreased nerve conduction velocitySeizureAtaxiaHyporeflexiaGait disturbanceMuscle weaknessProgressive spasticityFrequent fallsDevelopmental regressionIncreased CSF protein concentrationMuscle spasmFloppy infantPeripheral neuropathyHyperintensity of cerebral white matter on MRIUrinary incontinenceAtypical behaviorPsychosisEmotional labilityDementiaPersonality changesDysarthriaDystoniaTremorIncoordinationBowel incontinenceLimb painGastrostomy tube feeding in infancyFeeding difficultiesPainTip-toe gaitAddictive behaviorImpaired continenceSchizophreniaAbnormal duodenum morphologyIntussusceptionAbnormal stomach morphologyAbnormal gallbladder morphologyDecerebrate rigidityNeoplasm of the gallbladderHemobilia

Classification & Codes

MeSH Code

D007966

Orphanet Code

ORPHA:512
metachromatic leukodystrophy
MeSHD007966
OrphanetORPHA:512
Treatments0 drug(s)
Symptoms on record45 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO