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Ravicti
glycerol phenylbutyrate
Stapelingsziekten van ureumcyclus
✅ Geautoriseerd
✅
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Waarvoor het wordt gebruikt
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).
⚠️ Patiëntveiligheid
No
Officiële EMA-pagina
https://www.ema.europa.eu/en/medicines/human/EPAR/ravicti