X-linked adrenoleukodystrophy

ICD-10: E71.3MeSH: D000326ORPHA: 431 Treatment Available

Overview

peroxisomal disorder resulting in cerebral demyelination, axonal dysfunction in the spinal cord leading to spastic paraplegia, adrenal insufficiency and in some cases testicular insufficiency

Available Treatments (1)

DrugFormStatusCountriesLead Time
Lorenzo's oil
Oral liquid3

Clinical Presentation

Signs and symptoms associated with X-linked adrenoleukodystrophy, sourced from HPO and Orphanet clinical annotations.

Abnormality of visionVisual impairmentVisual lossAtypical behaviorDementiaHyperactivityIntellectual disabilityGait disturbanceSpecific learning disabilityProgressive hearing impairmentAbnormality of metabolism/homeostasisIncoordinationClumsinessHeadacheParaparesisSomatic sensory dysfunctionFunctional motor deficitAttention deficit hyperactivity disorderProgressive spastic paraparesisLeg muscle stiffnessCognitive impairmentNeurogenic bladderAggressive behaviorDisinhibitionAdrenal insufficiencyVisual field defectHemiparesisAphasiaIncreased intracranial pressureUrinary bladder sphincter dysfunctionIncreased circulating ACTH levelAbnormal sexual behaviorAbnormality of adrenal physiologyDiplopiaImpotenceParalysis

Classification & Codes

ICD-10 Code

E71.3

MeSH Code

D000326

Orphanet Code

ORPHA:43
X-linked adrenoleukodystrophy
ICD-10E71.3
MeSHD000326
OrphanetORPHA:43
Treatments1 drug(s)
Symptoms on record36 signs
Statuspublished

Treatment Summary

Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
X-linked adrenoleukodystrophy | OrphanDrug