Evox Therapeutics Limited products

Argininosuccinic aciduria (ASA) is a rare genetic disorder characterized by a deficiency or lack of the enzyme argininosuccinate lyase (ASL). 

The second urea cycle disorder indication to be targeted by Evox is Citrullinemia type I, which is caused by a mutation in the ASS1 gene leading to a deficiency in hepatic enzyme arginosuccinate synthetase (ASS) that results in high levels of citrulline and ammonia in the blood.

Phenylketonuria, or PKU, is an autosomal recessive inborn error of phenylalanine metabolism resulting in a deficiency of the hepatic enzyme phenylalanine hydroxylase (PAH).