Active substance
Treatment of non ketotic hyperglycinemia, urea cycle disorders including carbamoyl-phosphate synthase-1 deficiency, ornithine transcarbamylase deficiency, citrullinaemia type 1, argininosuccinic aciduria, hyperargininaemia, n-acetylglutamate synthase deficiency, ornithine translocase deficiency and lysinuric protein intolerance.
Values as published by each source: SFDA human drug list (4-2026, as published by the Council of Health Insurance; indications from the CHI Daman Drug Formulary), Drugs@FDA, EMA medicines data, Health Canada Drug Product Database (Open Government Licence – Canada), CMS NADAC, NHS Drug Tariff Part VIIIA (NHSBSA Copyright, Open Government Licence v3.0), WHO Model List of Essential Medicines (CC BY 3.0 IGO; regrouped by active substance, not created by WHO), EMA PRAC, US FDA safety-related labeling changes and MHRA Drug Safety Update (Open Government Licence v3.0). Sources and reuse. Not a substitute for the official registers or the product information.