Pyruvate Dehydrogenase Complex Deficiency Disease

Synonyms

Ataxia with Lactic Acidosis

Ataxia with Lactic Acidosis I

Ataxia with Lactic Acidosis, Type I

Ataxia, Intermittent, with Abnormal Pyruvate Metabolism

Ataxia, Intermittent, with Pyruvate Dehydrogenase, or Decarboxylase, Deficiency

Deficiency, PDH

Deficiency, PDHC

Deficiency, Pyruvate Decarboxylase

Deficiency, Pyruvate Dehydrogenase

Intermittent Ataxia with Pyruvate Dehydrogenase Deficiency

Juvenile Pyruvate Dehydrogenase Complex Deficiency Disease

Lactic Acidosis with Ataxia, Type I

Neonatal Pyruvate Dehydrogenase Complex Deficiency Disease

PDH Deficiency

PDHC Deficiency

PDHC Deficiency Disease

Pyruvate Decarboxylase Deficiency

Pyruvate Dehydrogenase Complex Deficiency

Pyruvate Dehydrogenase Complex Deficiency Disease, Juvenile

Pyruvate Dehydrogenase Complex Deficiency Disease, Neonatal

Pyruvate Dehydrogenase Deficiency

Type I Ataxia with Lactic Acidosis

An inherited metabolic disorder caused by deficient enzyme activity in the PYRUVATE DEHYDROGENASE COMPLEX, resulting in deficiency of acetyl CoA and reduced synthesis of acetylcholine. Two clinical forms are recognized: neonatal and juvenile. The neonatal form is a relatively common cause of lactic acidosis in the first weeks of life and may also feature an erythematous rash. The juvenile form presents with lactic acidosis, alopecia, intermittent ATAXIA; SEIZURES; and an erythematous rash. (From J Inherit Metab Dis 1996;19(4):452-62) Autosomal recessive and X-linked forms are caused by mutations in the genes for the three different enzyme components of this multisubunit pyruvate dehydrogenase complex. One of the mutations at Xp22.2-p22.1 in the gene for the E1 alpha component of the complex leads to LEIGH DISEASE.