Glycerol phenylbutyrate, also known as HPN-100, is under trials in the treatment of certain inborn urea cycle disorders. The medication works by preventing the harmful buildup of ammonia in the body. Ammonia is a neurotoxic agent that is primarily generated in the intestine and detoxified in the liver. Toxic increases in systemic ammonia levels predominantly result from an inherited or acquired impairment in hepatic detoxification and lead to potentially life-threatening neuropsychiatric symptoms.
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