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Ammonaps
sodium phenylbutyrate
Ornithine Carbamoyltransferase Deficiency Disease;Citrullinemia;Carbamoyl-Phosphate Synthase I Deficiency Disease
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Authorised
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No active alerts
This medicine has no ongoing shortages, safety letters or recalls.
What it's used for
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.
โ ๏ธ Patient safety
No
Official EMA page
https://www.ema.europa.eu/en/medicines/human/EPAR/ammonaps