Sodium phenylbutyrate is a salt of an aromatic fatty acid. The compound is used to treat urea cycle disorders, because its metabolites offer an alternative pathway to the urea cycle to allow excretion of excess nitrogen. Sodium phenylbutyrate is also a histone deacetylase inhibitor and chemical chaperone, leading respectively to research into its use as an anti-cancer agent and in protein misfolding diseases such as cystic fibrosis. It is used as adjunctive therapy for the management of chronic urea cycle disorders due to deficiencies in carbamylphosphate (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase. It is indicated in all neonatal- onset efficiency presenting within the first 28 days of life. Also indicated in patients with late-onset, presenting after the first month of life with a history of hyperammonemic encephalopathy. Sodium phenylbutyrate is a pro-drug and is rapidly metabolized to phenylacetate. Phenylacetate is a metabolically active compound that conjugates with glutamine via acetylation to form phenylacetylglutamine. The kidneys then excrete Phenylacetylglutamine. PBA (phenylbutyric acid) is absorbed from the intestine and converted by way of β-oxidation to the active moiety, phenylacetic acid (PAA). PAA is conjugated with glutamine in the liver and kidney by way of N-acyl coenzyme A-l-glutamine N-acyltransferase to form phenylacetylglutamine (PAGN). Like urea, PAGN incorporates two waste nitrogens and is excreted in the urine. On a molar basis, it is comparable to urea (each containing two moles of nitrogen). Therefore, phenylacetylglutamine provides an alternate vehicle for waste nitrogen excretion.

Phenylacetic acid (abr. PAA and synonyms are: α-toluic acid, benzeneacetic acid, alpha tolylic acid, 2-phenylacetic acid, β-phenylacetic acid) is an organic compound containing a phenyl functional group and acarboxylic acid functional group. Because it is used in the illicit production of phenylacetone (used in the manufacture of substituted amphetamines), it is subject to controls in countries including the United States and China Phenylacetic acid is used in some perfumes, possessing a honey-like odor in low concentrations, and is also used in penicillin G production. It is also employed to treat type II hyperammonemia to help reduce the amounts of ammonia in a patient's bloodstream by forming phenylacetyl-CoA, which then reacts with nitrogen-rich glutamine to form phenylacetylglutamine. This compound is then secreted by the patient's body. In Phase 2 of clinical research it investigated in the treatment of Brain and Central Nervous System Tumors.

Glycerin (glycerol) is 3-carbon alcohol naturally occurring in the human body. It is the structural backbone triacylglycerol molecules, and can also be converted to a glycolytic substrate for subsequent metabolism. Glycerin is a colorless, odorless, viscous, sweet-tasting liquid. The FDA classifies glycerol as "generally recognized as safe". Glycerin is used in the pharmaceutical industry as a sweetener in syrups, lozenges, and as an excipient in eyewash solutions. As an individual prescription product, glycerin has uses as a hyperosmotic, osmotic diuretic, and ophthalmic agent. It may be used as an eye drop in the treatment of glaucoma to reduce intraocular pressure, as a solution or suppository for short-term treatment of constipation, to evacuate the bowel prior to a colonoscopy, and in some ocular surgeries. It may be given intravenously to reduce pressure inside the brain and used externally on the skin as a moisturizer. Glycerin has many other uses in the agricultural, food and pharmaceutical industry.

Tributyrin is a prodrug of natural butyrate. It is a neutral short-chain fatty acid triglyceride that is likely to overcome the pharmacokinetic drawbacks of natural butyrate as a drug. Tributyrin has potent antiproliferative, proapoptotic and differentiation-inducing effects in neoplastic cells. Compared with butyrate, tributyrin has more favorable pharmacokinetics and is well tolerated. Because it is rapidly absorbed and chemically stable in plasma, tributyrin diffuses through biological membranes and is metabolized by intracellular lipases, releasing therapeutically effective butyrate over time directly into the cell. Tributyrin may, at least in part, exert its growth-reducing and differentiation-inducing effect in Caco-2 cells by an upregulation of the vitamin D receptor; this may provide a useful therapeutic approach in chemoprevention and treatment of colorectal cancer. In phase I study of the orally administered tributyrin there was no consistent increase in hemoglobin F. Peak plasma butyrate concentrations occurred between 0.25 and 3 h after dose. Development of tributyrin as an anticancer agent was discontinued.

1,2-DIMYRISTOYL-SN-GLYCERO-3-(PHOSPHO-S-(1-GLYCEROL)), SODIUM SALT is a prototype for the preparation of liposomes with negatively charged hydrophilic head groups. It can be used in the generation of different types of artificial membranes.