Carglumic Acid

Description: Carglumic Acid (Ureidoglutaric acid) is an orphan drug used to treat hyperammonemia in patients with N-acetyl glutamate synthase absence. This rare hereditary disease results in elevated blood levels of ammonia, which can eventually cross the blood-brain barrier and lead to neurologic problems, coma, cerebral edema, and death. Carglumic acid was approved by the U.S. FDA on 18 March 2010. Target: Others. Smiles: [C@@H](CCC(O)=O)(NC(N)=O)C(O)=O. References: Carglumic acid. Prescrire Int. 2008 Apr,17(94):50-1.