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Genomic newborn screening: ICoNS v1.12 ACAT1 Lilian Rudd gene: ACAT1 was added
gene: ACAT1 was added to Genomic newborn screening: ICoNS. Sources: Expert List
Mode of inheritance for gene: ACAT1 was set to BIALLELIC, autosomal or pseudoautosomal
Phenotypes for gene: ACAT1 were set to Alpha-methylacetoacetic aciduria, MIM#203750; Beta-ketothiolase deficiency MONDO:0008760
Added comment: Well established gene-disease association. DEFINITIVE by ClinGen.

Childhood onset, presents with metabolic acidosis.

Non-genetic confirmatory testing: urine organic acids, plasma acylcarnitine profile

Treatment: avoid fasting, carnitine, riboflavin, protein restricted diet
Sources: Expert List