251. 尿素サイクル異常症
［臨床試験数：42，薬物数：45（DrugBank：10），標的遺伝子数：4，標的パスウェイ数：29］

Searched query = "Urea cycle disorder", "N-acetylglutamate synthase deficiency", "NAGS deficiency", "Carbamoyl phosphate synthetase I deficiency", "CPS1 deficiency", "Ornithine transcarbamylase deficiency", "OTC deficiency", "Classic citrullinemia", "Citrullinemia type I", "Argininosuccinic aciduria", "Argininemia", "Hiperornitinemia-hiperamonemia-homocitrulinuria syndrome", "HHH syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 4-phenylbutyrate; ALLOGENIC HUMAN HETEROLOGOUS LIVER CELLS; ARCT-810; ASA; Acetohydroxamic Acid; Acetohydroxamic Acid Oral Tablet [Lithostat]; Acetohydroxamic acid; Arginine; Behavioral: Protein and calorie controlled diet; Carglumic acid; Celulas hepatocitos troncales adultas alogenicas de higado expandidas; DTX301; GT4P; Genetic: Ornithine transcarbamylase vector; Genetic: recombinant adenovirus containing the ornithine transcarbamylase gene; Genetic: scAAV8OTC; Glyceryl tri-(4-phenylbutyrate); Glyceryl tri-(4-phenylbutyrate)(GT4P); HHALPC; HHLivC; HPN-100; HepaStem; HepaStem Infusion; Heparesc; Heterologous Human Adult Liver-derived Progenitor Cells; Heterologous Human Adult Liver-derived Progenitor Cells (HHALPC); Human Heterologous Liver Cells; Human heterologous liver cells (for infusion); Isotopic Intravenous [13C]-Urea; KB195; MRT5201; NaPBA; Neo-ASA; Nitric Oxide; Nitric oxide supplement; Oral prednisone; Ornithine; Phenylbutyrate; Prednisone; Promethera HepaStem; RAVICTI; Ravicti; SYNB1020; Sodium Phenylbutyrate; Urea