Carglumic acid approved to treat genetic hyperammonemia

Thompson, Cheryl A.
May 2010
American Journal of Health-System Pharmacy;5/1/2010, Vol. 67 Issue 9, p690
Academic Journal
The article reports on the U.S. Food and Drug Administration's (FDA's) approval of carglumic acid or Carbaglu for genetic hyperammonemia treatment. Its manufacturer Orphan Europe notes that the drug resembles N-acetylglutamate to activate the first urea cycle enzyme and lessen the plasma ammonia level of N-acetylglutamate synthase deficient patients to normal. The FDA finds the drug effective in treating acute hyperammonemia due to the deficiency.


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