ornithine translocase deficiency

MeSH: C538380ORPHA: 415

Overview

amino acid metabolic disorder that has material basis in deficiency of ornithine translocase resulting in the accumulation of ammonia in the blood

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 ornithine translocase deficiency, sourced from HPO and Orphanet clinical annotations.

HyperreflexiaHyperammonemiaAbnormal circulating citrulline concentrationHyperornithinemiaNeurodevelopmental delayCognitive impairmentIntellectual disabilityLethargySpastic paraplegiaConfusionGeneralized hypotoniaSpecific learning disabilityDecreased liver functionFailure to thriveProtein avoidanceProgressive cerebellar ataxiaCerebral cortical atrophyClonusHepatomegalyIncoordinationImpaired vibratory sensationEpisodic vomitingTachypneaElevated circulating hepatic transaminase concentrationOroticaciduriaAcute encephalopathyAbnormal pyramidal signSpeech apraxiaFeeding difficultiesHepatitisSeizureComaRespiratory alkalosisSpastic gaitGeneralized myoclonic seizureAbnormality of the coagulation cascadeMultifocal cerebral white matter abnormalitiesChorioretinal atrophyHepatic failureChorioretinal hypopigmentation

Classification & Codes

MeSH Code

C538380

Orphanet Code

ORPHA:415
ornithine translocase deficiency
MeSHC538380
OrphanetORPHA:415
Treatments0 drug(s)
Symptoms on record40 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO