Synonyms: 4-phenylbutanoic acid | Ammonaps® | Buphenyl® | gamma-phenylbutyric acid | triButyrate
phenylbutyrate is an approved drug (FDA (1996), EMA (1999))
Compound class:
Synthetic organic
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No information available. |
Mechanism Of Action and Pharmacodynamic Effects ![]() |
In patients with liver enzyme deficiency hyperammonemia can develop due to impaired waste nitrogen removal. Sodium phenylbutyrate is converted to phenylacetate (active metabolite) which acts as an ammonia sink and chemical chaperone and prevents waste nitrogen from entering the urea cycle, thus preventing hyperammonemia. Sodium phenylbutyrate also acts via HDACs as a transcriptional regulator to alter chromatin structure [2]. This latter action is reported to provide neuroprotective effects [1], which may help reduce the progressive brain damage which is a major pathological symptom of metabolic disorders such as maple syrup urine disease, proteinuric diseases and other urea cycle disorders. In 5q spinal muscular atrophy, sodium phenylbutyrate increases transcription of the SMN2 (survival of motor neuron 2) gene via inhibition of histone deacetylase activity thus effecting elevated levels of functional SMN2 protein [2]. |