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Ammonaps
sodium phenylbutyrate
Deficiență de ornitină carbamoiltransferază;Citrulinemie;Deficiență de carbamoil-fosfat sintetază I
✅ Autorizat
✅
Nu există alerte active
Acest medicament nu are penurii în curs, scrisori de siguranță sau retrageri de pe piață.
La ce se utilizează
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.
⚠️ Siguranță a pacientului
No
Pagina oficială EMA
https://www.ema.europa.eu/en/medicines/human/EPAR/ammonaps