Evidence supporting the use of: Arginine carglumate
For the body system: Hepatic System
Synopsis
Source of validity: Scientific
Rating (out of 5): 5
Arginine carglumate is scientifically validated for use in supporting the hepatic system, particularly in the treatment of hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency and certain organic acidurias. It is a structural analogue of N-acetylglutamate (NAG), an essential activator of carbamoyl phosphate synthetase I (CPS1), the first enzyme of the urea cycle, which takes place in the liver. In conditions where NAG is deficient, such as NAGS deficiency, the urea cycle cannot properly detoxify ammonia, leading to dangerous elevations in blood ammonia levels (hyperammonemia). Carglumic acid (arginine carglumate) restores CPS1 activity by mimicking NAG, thereby facilitating ammonia detoxification in the liver. Clinical studies and regulatory approvals (such as by the EMA and FDA) support its efficacy and safety in these rare but severe hepatic metabolic disorders. While its use is not for general liver support, it is critical in the management of specific inherited metabolic diseases that impair hepatic ammonia metabolism. Multiple clinical trials, case studies, and consensus guidelines confirm its role as a life-saving therapy in these contexts. Therefore, its application in the hepatic system is grounded in strong scientific evidence and is considered standard of care in relevant metabolic conditions.
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