We provide you with the best medicine purchasing service
Quickly morph client-centric results through performance based applications. Proactively facilitate professional human capital for cutting-edge.
Download Now
N-carbamoylglutamate (Carbaglu®)
Unknown hyperammonemia, NAGS deficiency, CPS-1 deficiency, propionic acidemia or methylmalonic acidemia.
100-250 mg/kg/day then adjusted individually in order to maintain normal ammonia plasma levels and divided into 2 to 4 doses (130-137).
Base on limited unpublished data the maintenance dose less than 100 mg/kg/day.
200 mg tablet
PO
Gastrointestinal: Abdominal pain (17% ), Diarrhea (13% ), Vomiting (26% )
Hematologic: Anemia (13% ), Decreased hemoglobin (13% )
Immunologic: Infectious disease (13% )
Neurologic: Headache (13% )
Otic: Infection of ear (13% )
Respiratory: Nasopharyngitis (13% ), Tonsillitis (17% )
Other: Fever (17%)
4
130. Cox T, Lachmann R, Hollak C, et al. Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. Lancet 2000;355(9214):1481-5 doi: 10.1016/S0140-6736(00)02161-9[published Online First: Epub Date]|.
131. Pastores GM, Barnett NL, Kolodny EH. An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment. Clinical therapeutics 2005;27(8):1215-27 doi: 10.1016/j.clinthera.2005.08.004[published Online First: Epub Date]|.
132. Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P. Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. American journal of hematology 2005;80(3):223-9 doi: 10.1002/ajh.20504[published Online First: Epub Date]|.
133. Zimran A, Elstein D. Gaucher disease and the clinical experience with substrate reduction therapy. Philosophical transactions of the Royal Society of London. Series B, Biological sciences 2003;358(1433):961-6 doi: 10.1098/rstb.2003.1272[published Online First: Epub Date]|.
134. Patterson MC, Vecchio D, Jacklin E, et al. Long-term miglustat therapy in children with Niemann-Pick disease type C. J Child Neurol 2010;25(3):300-5 doi: 10.1177/0883073809344222[published Online First: Epub Date]|.
135. Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE. Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet neurology 2007;6(9):765-72 doi: 10.1016/S1474-4422(07)70194-1[published Online First: Epub Date]|.
136. Guffon N, Vianey-Saban C, Bourgeois J, Rabier D, Colombo JP, Guibaud P. A new neonatal case of N-acetylglutamate synthase deficiency treated by carbamylglutamate. J Inherit Metab Dis 1995;18(1):61-5
137. Haberle J. Role of carglumic acid in the treatment of acute hyperammonemia due to N-acetylglutamate synthase deficiency. Ther Clin Risk Manag 2011;7:327-32 doi: 10.2147/TCRM.S12703[published Online First: Epub Date]|.
