Pyruvate dehydrogenase E1-beta deficiency

MeSH: C566729ORPHA: 255138

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 E1-beta deficiency, sourced from HPO and Orphanet clinical annotations.

HypotoniaAgenesis of corpus callosumAbnormal facial shapeAbnormal calvaria morphologyDecreased activity of the pyruvate dehydrogenase complexLactic acidosisDecreased body weightNeurodevelopmental delaySeizureAtaxiaPachygyriaReduced tendon reflexesCerebellar hypoplasiaIntrauterine growth retardationHypoplasia of the corpus callosumVentriculomegalyHypoplasia of the brainstemPeriventricular leukomalaciaCorticospinal tract hypoplasiaPeriventricular cystsPeriventricular heterotopiaImpaired smooth pursuitShort corpus callosum

Classification & Codes

MeSH Code

C566729

Orphanet Code

ORPHA:255138
Pyruvate dehydrogenase E1-beta deficiency
MeSHC566729
OrphanetORPHA:255138
Treatments0 drug(s)
Symptoms on record23 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO
Pyruvate dehydrogenase E1-beta deficiency | OrphanDrug