Carbamoyl-phosphate synthetase 1 deficiency
ORPHA:147· ICD-10 E72.2
Definition
A rare, severe disorder of urea cycle metabolism typically characterized by either a neonatal-onset of severe hyperammonemia that occurs few days after birth and manifests with lethargy, vomiting, hypothermia, seizures, coma and death or a presentation outside the newborn period at any age with (sometimes) milder symptoms of hyperammonemia.
- Prevalence
- Unknown
- Inheritance
- Autosomal recessive
- Age of onset
- All ages