FDA clears drug for urea cycle disorders
The Food and Drug Administration recently approved glycerol phenylbutyrate for the chronic management of some urea cycle disorders in patients aged 2 years and older. A UCD is a genetic disorder caused by a mutation that results in a deficiency of 1 of the 6 enzymes in the urea cycle. The deficiency ultimately leads to a buildup of ammonia in the blood, which can be fatal. According to the National Urea Cycle Disorders Foundation, the estimated incidence of UCDs is 1 in 8500 births.
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