non-progressive predominantly posterior cavitating leukoencephalopathy with peripheral neuropathy

ORPHA: 4362711 Treatment Available

Overview

human disease

Available Treatments (1)

DrugFormStatusCountriesLead Time
gabapentin
Oral capsule 100mg, 300mg, 400mg; Oral tablet 600mg, 800mg; Oral solution 250mg/5mLFDA Approved, EMA Approved113d

Clinical Presentation

Signs and symptoms associated with non-progressive predominantly posterior cavitating leukoencephalopathy with peripheral neuropathy, sourced from HPO and Orphanet clinical annotations.

Progressive leukoencephalopathyProgressive peripheral neuropathyProteinuriaRenal tubular dysfunctionPtosisPigmentary retinopathyOptic atrophyDelayed speech and language developmentIntellectual disabilitySeizureAtaxiaExcessive daytime somnolenceGlobal developmental delayMotor delayGait disturbanceGeneralized hypotoniaDecreased liver functionFailure to thriveHypertrophic cardiomyopathyAnemiaRenal Fanconi syndromeHepatomegalyDevelopmental regressionIncreased CSF lactateRespiratory insufficiency due to muscle weaknessExertional dyspneaGlycosuriaHyperphosphaturiaLactic acidosisGeneralized muscle weaknessAminoaciduriaDiffuse hepatic steatosisAbnormal pyramidal signBilateral sensorineural hearing impairmentFatigable weakness of swallowing musclesSkeletal muscle steatosisSpastic tetraparesisVomiting

Classification & Codes

Orphanet Code

ORPHA:436271
non-progressive predominantly posterior cavitating leukoencephalopathy with peripheral neuropathy
OrphanetORPHA:436271
Treatments1 drug(s)
Symptoms on record38 signs
Statuspublished

Treatment Summary

Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO