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J Pediatr ; 127(5): 723-8, 1995 Nov.
Artículo en Inglés | MEDLINE | ID: mdl-7472823

RESUMEN

Carnitine-acylcarnitine translocase deficiency is a newly recognized inborn error of metabolism that involves transport of long-chain fatty acids into mitochondria, which in turn impairs mitochondrial beta-oxidation, and ketogenesis. We report a new familial example; the affected twins had neonatal distress, hyperammonemia, and transient intracardiac conduction defects. Clinical and biochemical analysis of both our patients and the two previously reported patients revealed that this inherited defect could be manifested during the neonatal period without any of the signs classically associated with fatty oxidation defects. In contrast, all four patients had sustained and "isolated" hyperammonemia, which could be misinterpreted as being caused by urea cycle defects. We conclude that carnitine-acylcarnitine translocase deficiency is a potential differential diagnosis in neonates with unexplained neonatal hyperammonemia. Cardiac and muscle involvement may represent further early pivotal symptoms.


Asunto(s)
Amoníaco/sangre , Carnitina Aciltransferasas/deficiencia , Enfermedades en Gemelos/diagnóstico , Errores Innatos del Metabolismo Lipídico/diagnóstico , Carnitina Aciltransferasas/análisis , Diagnóstico Diferencial , Enfermedades en Gemelos/etiología , Enfermedades en Gemelos/genética , Resultado Fatal , Femenino , Humanos , Recién Nacido , Recien Nacido Prematuro , Errores Innatos del Metabolismo Lipídico/sangre , Errores Innatos del Metabolismo Lipídico/etiología , Errores Innatos del Metabolismo Lipídico/genética
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