It treats urea cycle disorders, genetic diseases in which nitrogen waste builds up in the blood plasma as ammonia glutamine (a state called hyperammonemia) due to deficiences in the enzymes carbamoyl phosphate synthetase I, ornithine transcarbamylase, or argininosuccinic acid synthetase. Uncontrolled, this causes intellectual impairment and early death.
Urea cycle disorders, genetic diseases characterized by nitrogen waste accumulation in blood plasma as ammonia, due to enzyme deficiencies in carbamoyl phosphate synthetase I, ornithine transcarbamylase, or argininosuccinic acid synthetase. Uncontrolled, it leads to intellectual impairment and early death.