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Ammonaps
sodium phenylbutyrate
Deficiencia de ornitina carbamoiltransferasa; Citrulinemia; Deficiencia de carbamoil-fosfato sintetasa I
✅ Autorizado
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Este medicamento no tiene escasez en curso, cartas de seguridad ni retiradas del mercado.
Para qué se utiliza
Ammonaps is indicated as adjunctive therapy in the chronic management of urea cycle disorders, involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase orargininosuccinate synthetase. It is indicated in all patients with neonatal-onset presentation (complete enzyme deficiencies, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease(partial enzyme deficiencies, presenting after the first month of life) who have a history of hyperammonaemic encephalopathy.
⚠️ Seguridad del paciente
No
Página oficial de la EMA
https://www.ema.europa.eu/en/medicines/human/EPAR/ammonaps