Arginine:glycine amidinotransferase (AGAT) deficiency in a newborn: early treatment can prevent phenotypic expression of the disease.

Battini, Roberta; Alessandrì, M Grazia; Leuzzi, Vincenzo; Moro, Francesca; Tosetti, Michela; Bianchi, Maria C; Cioni, Giovanni · J Pediatr · 2006

case_report · Level V

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Abstract

Arginine:glycine amidinotransferase deficiency is a treatable inborn error of creatine synthesis, characterized by mental retardation, language impairment, and behavioral disorders. We describe a patient in whom arginine:glycine amidinotransferase was diagnosed at birth and treated at 4 months with creatine supplementation. In contrast with his 2 older sisters, he had normal psychomotor development at 18 months.

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