Title |
New developments in the treatment of hyperammonemia: emerging use of carglumic acid
|
---|---|
Published in |
International Journal of General Medicine, January 2011
|
DOI | 10.2147/ijgm.s10490 |
Pubmed ID | |
Authors |
Marta Daniotti, Giancarlo la Marca, Patrizio Fiorini, Luca Filippi |
Abstract |
Hyperammonemia is a true neonatal emergency with high toxicity for the central nervous system and developmental delay. The causes of neonatal hyperammonemia are genetic defects of urea cycle enzymes, organic acidemias, lysinuric protein intolerance, hyperammonemia-hyperornithinemia- homocitrullinemia syndrome, transient hyperammonemia of the newborn, and congenital hyperinsulinism with hyperammonemia. In some of these conditions the high blood ammonia levels are due to the reduction of N-acetylglutamate, an essential cofactor necessary for the function of the urea cycle, or to the reduction of carbamoyl-phosphate synthase-I activity. In these cases, N-carbamylglutamate (carglumic acid) can be administered together with the conventional therapy. Carglumic acid is an analog of N-acetylglutamate that has a direct action on carbamoyl-phosphate synthase-I. Its effects are reactivation of the urea cycle and reduction of plasma ammonia levels. As a consequence it improves the traditional treatment, avoiding the need of hemodialysis and peritoneal dialysis. In this review we evaluate the possible field of application of carglumic acid and its effectiveness and safety. |
X Demographics
As of 1 July 2024, you may notice a temporary increase in the numbers of X profiles with Unknown location. Click here to learn more.
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 2 | 100% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 2 | 100% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
United Kingdom | 1 | 2% |
Spain | 1 | 2% |
Colombia | 1 | 2% |
Unknown | 58 | 95% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Researcher | 13 | 21% |
Other | 10 | 16% |
Student > Ph. D. Student | 7 | 11% |
Student > Master | 6 | 10% |
Student > Bachelor | 5 | 8% |
Other | 10 | 16% |
Unknown | 10 | 16% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 23 | 38% |
Agricultural and Biological Sciences | 6 | 10% |
Pharmacology, Toxicology and Pharmaceutical Science | 5 | 8% |
Biochemistry, Genetics and Molecular Biology | 4 | 7% |
Psychology | 3 | 5% |
Other | 7 | 11% |
Unknown | 13 | 21% |