Glycerol phenylbutyrate
未知生产厂商
Glycerol phenylbutyrate is a nitrogen-binding agent used to manage patients with urea cycle disorders who cannot be sufficiently managed amino acid supplementation and/or dietary restrictions.
Glycerol phenylbutyrate is a nitrogen-binding agent used to manage patients with urea cycle disorders who cannot be sufficiently managed amino acid supplementation and/or dietary restrictions.
The toxic accumulation of ammonia in the blood and brain arise from urea cycle disorders in which patients are deficient in critical enzymes or transporters that are involved in the synthesis of urea from ammonia. Glycerol phenylbutyrate is a prodrug - the major metabolite, phenylacetate (PAA) is the molecule that binds to nitrogen. PAA conjugates with glutamine (which contains 2 molecules of nitrogen) via acetylation in the liver and kidneys to form phenylacetylglutamine (PAGN), which is excreted by the kidneys. PAGN, like urea, contains 2 moles of nitrogen and provides an alternate vehicle for waste nitrogen excretion.
Glycerol phenylbutyrate is a nitrogen-binding agent for the chronic management of adult and pediatric patients ≥2 years of age with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
共 1 项相关疾病
