4H leukodystrophy

ORPHA: 289494

Overview

rare 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 4H leukodystrophy, sourced from HPO and Orphanet clinical annotations.

Hypogonadotropic hypogonadismAbnormality of the dentitionMyopiaHypodontiaAtaxiaDysarthriaDystoniaCerebral hypomyelinationAbnormality of eye movementDelayed pubertyTremorDysphagiaAbnormality of extrapyramidal motor functionDroolingUpper motor neuron dysfunctionShort statureProgressive gait ataxiaHyperintensity of cerebral white matter on MRIAbnormal saccadic eye movementsGaze-evoked nystagmusDelayed eruption of teethDecreased response to growth hormone stimulation testSeizureMental deteriorationCerebellar atrophyDysmetriaDysdiadochokinesisHypoplasia of the corpus callosumAbnormality of thyroid physiologyCataractOptic atrophyStriatal T2 hyperintensity

Classification & Codes

Orphanet Code

ORPHA:289494
4H leukodystrophy
OrphanetORPHA:289494
Treatments0 drug(s)
Symptoms on record32 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO