Pyruvate Carboxylase Deficiency

Alternative Names

  • PC Deficiency
  • Ataxia with Lactic Acidosis II
  • Leigh Necrotizing Encephalopathy due to Pyruvate Carboxylase Deficiency
  • Leigh Syndrome due to Pyruvate Carboxylase Deficiency

Associated Genes

Pyruvate Carboxylase
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WHO-ICD-10 version:2010

Endocrine, nutritional and metabolic diseases

Metabolic disorders

OMIM Number

266150

Mode of Inheritance

Autosomal recessive

Gene Map Locus

11q13.2

Description

PC deficiency may be categorized into 3 phenotypic subgroups. Patients from North America ('group A') have lactic acidemia and psychomotor retardation, whereas those from France and the United Kingdom ('group B') have a more complex biochemical phenotype with increased serum lactate, ammonia, citrulline, and lysine, as well as an intracellular redox disturbance in which the cytosolic compartment is more reduced and the mitochondrial compartment is more oxidized. Patients in group B have decreased survival compared to group A, and usually do not survive beyond 3 months of age. Group C is relatively benign. [From OMIM]

Epidemiology in the Arab World

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Subject IDCountrySexFamily HistoryParental ConsanguinityHPO TermsVariantZygosityMode of InheritanceReferenceRemarks
266150.1Saudi ArabiaFemaleYesYes Abnormal cerebral white matter morpholog...NM_001040716.2:c.1480C>THomozygousAutosomal, RecessiveMonies et al. 2017
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