galactokinase deficiency

ORPHA: 79237

Overview

galactosemia that involves an accumulation of galactose and galactitol secondary to the decreased conversion of galactose to galactose-1-phosphate by galactokinase

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 galactokinase deficiency, sourced from HPO and Orphanet clinical annotations.

HypergalactosemiaAbnormal circulating enzyme concentration or activityIncreased level of galactitol in plasmaIncreased level of galactitol in urineCataractNuclear cataractHypergonadotropic hypogonadismHyperinsulinemiaIntellectual disabilityMotor delayHepatosplenomegalySmall for gestational ageHepatomegalyPremature ovarian insufficiencySpeech apraxiaMicrocephalySensorineural hearing impairmentSeizureFailure to thrivePremature birthHypoglycemiaPsychomotor deteriorationHypercholesterolemiaReduced circulating complement concentrationFeeding difficultiesNeonatal asphyxia

Classification & Codes

Orphanet Code

ORPHA:79237
galactokinase deficiency
OrphanetORPHA:79237
Treatments0 drug(s)
Symptoms on record26 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
galactokinase deficiency | OrphanDrug