Metabolically stable analog of N-acetylglutamate, a physiological activator of the first enzyme of the urea cycle, carbamylphosphate synthetase (CAPS). Prepn: H. McIlwain, Biochem. J. 33, 1942 (1939). Effect on blood urea and ammonia levels and potential clinical application: J.-E. O'Connor et al., Eur. J. Pediatr. 143, 196 (1985). Evaluation in treatment of CAPS deficiency: G. Kuchler et al., J. Inherit. Metab. Dis. 19, 220 (1996); of N-acetylglutamate synthetase (NAGS) deficiency: B. Plecko et al., Eur. J. Pediatr. 157, 996 (1998).
In treatment of inherited urea cycle disorders.