Pyruvate dehydrogenase E2 deficiency

MeSH: C565448ORPHA: 79244

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 Pyruvate dehydrogenase E2 deficiency, sourced from HPO and Orphanet clinical annotations.

Gait disturbanceAbnormal circulating enzyme concentration or activityDecreased circulating vitamin B1 concentrationAbnormal CSF pyruvate family amino acid concentrationAbnormality of eye movementRetinal degenerationAbnormality of the nervous systemAtypical behaviorDysarthriaGlobal developmental delayHypertoniaBroad-based gaitNeurodegenerationParoxysmal dystoniaLower limb hyperreflexiaEye of the tiger anomaly of globus pallidusBabinski signPositional foot deformitySevere intellectual disabilityUpgaze palsyDelayed ability to standFrog-leg postureDelayed ability to walkArm dystoniaMicrocephalyStrabismusDementiaAnxietyFunctional motor deficitPeripheral visual field lossSpeech apraxia

Classification & Codes

MeSH Code

C565448

Orphanet Code

ORPHA:79244
Pyruvate dehydrogenase E2 deficiency
MeSHC565448
OrphanetORPHA:79244
Treatments0 drug(s)
Symptoms on record31 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO