This open-label, uncontrolled, 25-year study of treatment with sodium phenylacetate and sodium benzoate documents overall survival of 84% in historically lethal urea-cycle disorders characterized by acute hyperammonemic episodes. When episodes are promptly recognized and treated with this therapy, together with intravenous arginine hydrochloride and adequate calories to prevent catabolism, plus dialysis when necessary, plasma ammonium levels fall, markedly improving survival.
More...