Carglumic Acid

From Anvita Health Wiki

Contents 1 Indications 2 Dosage 3 Monitor 4 Adverse-effects 5 Mechanism-of-action 6 More General Terms 7 References

Indications

• treatment of N-acetylglutamate synthase deficiency

Dosage

• initial dose of 100-250 mg/kg/day in 2-4 divided doses
• adjust to maintain normal ammonia plasma levels
• maintenance doses range from 10-100 mg/kg/day
• concomitant use of other ammonia-lowering therapies is recommended during episodes of acute hyperammonemia

Monitor

• liver function tests
• renal function tests
• complete blood count ( CBC)

Adverse-effects

• vomiting
• abdominal pain
• diarrhea
• fever
• tonsillitis
• anemia
• ear infection
• headache

Mechanism-of-action

• structural analogue of N-acetylglutamate, the naturally occurring activator of carbamoyl phosphate synthetase, the 1st enzyme of the urea cycle

More General Terms

• orphan drug
• metabolic agent (metabolic modifier)

References

1. Yael Waknine FDA Approves Carglumic Acid Therapy for Rare Genetic Disorder Medscape Today, March 19, 2010 [1]

carglumic acid (Carbaglu)