neuronal ceroid lipofuscinosis 8 northern epilepsy variant

ORPHA: 19471 Treatment Available

Overview

human disease

Available Treatments (1)

DrugFormStatusCountriesLead Time
brivaracetam
Orphan
oral tablet, 10mg, 25mg, 50mg, 75mg, 100mg; oral solution, 10mg/mL; IV injection, 50mg/5mLFDA Approved810d

Clinical Presentation

Signs and symptoms associated with neuronal ceroid lipofuscinosis 8 northern epilepsy variant, sourced from HPO and Orphanet clinical annotations.

Gastrostomy tube feeding in infancyT2 hypointense thalamusHyperintensity of cerebral white matter on MRIVisual impairmentIntellectual disabilitySeizureCerebellar atrophyInability to walkIntracellular accumulation of autofluorescent lipopigment storage materialVascular granular osmiophilic material depositionProgressive visual lossOptic disc pallorUndetectable electroretinogramDelayed speech and language developmentAtaxiaCerebral atrophyGait ataxiaGeneralized myoclonic seizureAbnormal speech patternClumsinessUnsteady gaitMotor deteriorationLoss of speechSpastic tetraplegiaCurvilinear intracellular accumulation of autofluorescent lipopigment storage materialFingerprint intracellular accumulation of autofluorescent lipopigment storage materialDifficulty standingEEG with generalized epileptiform dischargesEEG with abnormally slow frequenciesAbnormality of pattern visual evoked potentialsMotor regressionCognitive impairmentAtypical behaviorDementiaAutistic behaviorGlobal developmental delayDysphagiaFrequent fallsFocal impaired awareness seizure

Classification & Codes

Orphanet Code

ORPHA:1947
neuronal ceroid lipofuscinosis 8 northern epilepsy variant
OrphanetORPHA:1947
Treatments1 drug(s)
Symptoms on record39 signs
Statuspublished

Treatment Summary

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