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Ravicti
glycerol phenylbutyrate
Troubles du cycle de l'urée, congénitaux
✅ 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
Ravicti is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) including deficiencies of carbamoyl phosphate-synthase-I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. Ravicti must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).
⚠️ Sécurité des patients
No
Page officielle de l'EMA
https://www.ema.europa.eu/en/medicines/human/EPAR/ravicti