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. 2013 Aug;109(4):397-401.
doi: 10.1016/j.ymgme.2013.05.018. Epub 2013 Jun 6.

Use of carglumic acid in the treatment of hyperammonaemia during metabolic decompensation of patients with propionic acidaemia

M Abacan  1 , A Boneh
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Use of carglumic acid in the treatment of hyperammonaemia during metabolic decompensation of patients with propionic acidaemia

M Abacan et al. Mol Genet Metab. 2013 Aug.

Abstract

Propionic acidaemia (PA) results from propionyl-CoA carboxylase deficiency. During metabolic decompensation, the accumulation of propionyl-CoA causes secondary hyperammonaemia through N-acetylglutamate synthetase inactivation. Carglumic acid, a structural analogue of N-acetylglutamate, was given to patients with PA (n=3) during episodes of metabolic decompensation (n=8; age range: birth to 4years), in addition to high energy/low protein intake and carnitine. Plasma ammonia concentrations normalised within 6-19h. Carglumic acid was well tolerated with no side effects noted.

Keywords: Carglumic acid; Hyperammonaemia; Propionic acidaemia.

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