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Ravicti
glycerol phenylbutyrate
Ureacyklusforstyrrelser, medfødte
✅ Autoriseret
✅
Ingen aktive alarmer
Denne medicin har ingen igangværende mangel, sikkerhedsbreve eller tilbagekaldelser.
Hvad det bruges til
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).
⚠️ Patient sikkerhed
No
Officiel EMA-side
https://www.ema.europa.eu/en/medicines/human/EPAR/ravicti