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Beta ketothiolase deficiency MeSH Supplementary Concept Data 2026
An autosomal recessive inborn error of ISOLEUCINE catabolism characterized by urinary excretion of 2-methyl-3-hydroxybutyric acid, 2-methylacetoacetic acid, tiglylglycine, and 2-butanone. Some patients may experience KETOSIS during infections and some may have INTELLECTUAL DISABILITY. Mutations in the ACAT1 gene have been identified. OMIM: 203750