Glycerol phenylbutyrate

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Glycerol Phenylbutyrate

Glycerol phenylbutyrate (pronunciation: gly-SER-ol fen-il-BYOO-ti-rate) is a medication used in the treatment of urea cycle disorders.

Etymology

The term "glycerol phenylbutyrate" is derived from its chemical structure. "Glycerol" refers to the three-carbon alcohol base of the molecule, "phenyl" refers to the presence of a phenyl group, and "butyrate" refers to the presence of a butyric acid group.

Usage

Glycerol phenylbutyrate is used to manage and reduce the frequency of acute episodes of hyperammonemia in adults and children with urea cycle disorders. These disorders are a group of rare genetic conditions that result in a deficiency of one of the six enzymes in the urea cycle. This deficiency leads to an accumulation of ammonia in the blood, which can cause serious health problems.

Mechanism of Action

Glycerol phenylbutyrate works by providing an alternate pathway for the removal of excess nitrogen. It is metabolized in the body to phenylbutyrate, which is then converted into phenylacetylglutamine - a compound that can be excreted in the urine. This process helps to remove excess nitrogen and reduce the levels of ammonia in the blood.

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