CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders
Pharmaceutical Technology
JULY 29, 2022
However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. In 2013, the US Food and Drug Administration (FDA) approved Ravicti (glycerol phenylbutyrate), manufactured by Hyperion Therapeutics, which has since then been acquired by Horizon Pharma.
Let's personalize your content