Ornithine transcarbamylase deficiency is one of several rare diseases that have no approved drugs. This rare genetic disorder, which is more common in boys, arises from a deficiency of the critical OTC enzyme in liver cells, leading to dangerously elevated ammonia levels in the bloodstream. The consequences of uncontrolled ammonia buildup can be devastating, including impaired cognitive function, seizures, coma, and potentially life-threatening complications.
The stock we are bringing to your attention is that of a clinical-stage messenger RNA medicines company developing a drug for Ornithine transcarbamylase deficiency - Arcturus Therapeutics Holdings Inc. (ARCT).
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