metachromatic leukodystrophy, late infantile form

ORPHA: 309256

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, late infantile form, sourced from HPO and Orphanet clinical annotations.

Urinary incontinenceOptic atrophyDecreased nerve conduction velocitySeizureDysarthriaHyporeflexiaGeneralized hypotoniaMuscle weaknessDystoniaGait ataxiaClumsinessFrequent fallsDevelopmental regressionLeukodystrophyIncreased CSF protein concentrationEMG: chronic denervation signsProgressive peripheral neuropathyProgressive gait ataxiaTip-toe gaitPunctate periventricular T2 hyperintense fociAbnormality of visual evoked potentialsEmotional labilityHallucinationsDelusionCholecystitisSpasticityAbnormality of metabolism/homeostasisLoss of speechAbdominal distentionBabinski signReduced visual acuityBilateral sensorineural hearing impairmentFeeding difficulties in infancyAbnormal social behaviorDecerebrate rigidityVegetative state

Classification & Codes

Orphanet Code

ORPHA:309256
metachromatic leukodystrophy, late infantile form
OrphanetORPHA:309256
Treatments0 drug(s)
Symptoms on record36 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO