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Urea Cycle Disorder Equipment & Supplies
2 equipment items found
Manufactured by:Evox Therapeutics Limited based inOxford, UNITED KINGDOM
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 ...
by:Cartesian Therapeutics based inGaithersburg, MASSACHUSETTS (USA)
Selecta’s wholly owned gene therapy program for the treatment of ornithine transcarbamylase (OTC) deficiency is expected to enter the clinic in 2022. OTC deficiency is a genetic disorder urea cycle that causes ammonia to accumulate in the blood. The most severe form of the disorder presents within the first few days of ...