transketolase deficiency

ORPHA: 488618

Overview

human 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 transketolase deficiency, sourced from HPO and Orphanet clinical annotations.

Global developmental delayAbnormal heart morphologyProportionate short statureElevated circulating ribitol concentrationCataractUveitisCompulsive behaviorsDelayed speech and language developmentHypotoniaMild intellectual disabilityAbsent speechVentricular septal defectAtrial septal defectAttention deficit hyperactivity disorderIncreased level of ribose in urineRenal cystHearing impairmentConjunctivitisMotor stereotypySecondary amenorrheaSeborrheic dermatitisPatent ductus arteriosusPatent foramen ovaleHepatomegalyAbnormal coronary artery courseType I diabetes mellitusSelf-injurious behavior

Classification & Codes

Orphanet Code

ORPHA:488618
transketolase deficiency
OrphanetORPHA:488618
Treatments0 drug(s)
Symptoms on record27 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO