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Ammonaps
sodium phenylbutyrate
Déficit en ornithine carbamoyltransférase;Citrullinémie;Déficit en carbamoyl-phosphate synthase I
✅ Autorisé
✅
Aucune alerte active
Ce médicament ne fait l'objet ni de pénuries en cours, ni de lettres de sécurité, ni de rappels.
À quoi ça sert
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.
⚠️ Sécurité des patients
No
Page officielle de l'EMA
https://www.ema.europa.eu/en/medicines/human/EPAR/ammonaps