隐形杀手尿素循环障碍的克星来了！苯丁酸钠重磅上市！

Launched in China in May 2021, the drug is used to treat urea cycle disorders and can be used as an adjunct to dietary protein restriction and essential amino acid supplementation for the long-term management of patients with urea cycle disorders, including arginase deficiency (Argininemia), carbamoyl phosphate synthetase deficiency (CPS 1) and ornithine carbamoyltransferase deficiency (OTC) deficiency. It provides patients and clinicians with new medication options.

The dangers of urea cycle disorders

Urea cycle disorder refers to the patient's inability to eliminate ammonia produced by protein decomposition due to a lack of certain enzymes during the urea cycle. Excessive ammonia accumulates in the body, causing neurotoxicity and clinically manifesting as severe brain dysfunction.

The urea cycle function of some adult patients is barely able to maintain daily life, so they do not know that they have this disease, which means that their lives will be in danger once they become ill or have some accidents with their body.

Foreign media reported that the 25-year-old Hefford is strong and has a fitness habit. He will strictly control his diet and supplement a lot of protein. However, recently he has been drowsy and unable to cheer up. At first, Hefford didn't pay attention, thinking it was just a period of fatigue caused by fitness.

That was until Hefford collapsed in the gym and was declared brain dead by doctors the next day after being admitted to the hospital. It was only then that doctors diagnosed her with a urea cycle disorder. The large amount of protein Hefford usually consumed was broken down into ammonia. The large amount of ammonia accumulated in the body and caused toxicity, damaging his brain nerves.

Questions about sodium phenylbutyrate

Sodium phenylbutyrate is a drug that treats urea cycle disorders. Whether its mechanism of action can target this disease and whether it is clinically effective are issues of concern to many patients. The editor has sorted out the mechanism of action and efficacy of sodium phenylbutyrate for everyone. I hope it will be helpful to everyone.

The mechanism and efficacy of sodium phenylbutyrate

This drug is a prodrug of phenyl acetate. It is metabolized into phenyl acetate through beta-oxidation in the patient's body. Phenyl acetate is then conjugated with glutamine to form phenylacetylglutamine. The above two-step reactions are both fast and complete. Phenylacetyl glutamine, as a substitute for waste ammonia product of urea, can be excreted through the kidneys. Compared with urea at the molar level, each mole of both contains 2 moles of nitrogen (that is, 1 mole of this drug can also remove 2 moles of ammonia derived from amino acids). Effectively reduce the patient's amino acid levels.

Experiments have proven that oral administration of sodium phenylbutyrate at a dose of 27g/day can well achieve plasma concentrations that produce biological effects. In other experimental studies, nearly 25% of patients have been stable for more than six months. The treatment effect is remarkable.

Conclusion

Protein is an essential nutrient for the human body, but when protein is decomposed, ammonia is produced. If it is not decomposed in time, it will cause brain nerve poisoning, posing a great threat to patients with urea cycle disorders. Therefore, sodium phenylbutyrate is a rare and good medicine for treating urea cycle disorders due to its highly targeted mechanism of action and outstanding curative effect. If you need to purchase it, please consult your medical companion.

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