Carglumic acid

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Contents

Introduction

In the potentially fatal hyperammonaemia due to N-acetylglutamate synthase deficiency it will induce a rapid normalisation of plasma ammonia levels and indeed when treatment is started before any permanent brain damage, patients exhibited normal growth and psychomotor development. Structural analogue of N-acetylglutamate, the usual activator of carbamoyl phosphate synthetase, the first enzyme of the urea cycle.

Clinical Use

Indications

Administration

Oral usually starting dose 100 mg/kg, increasing if necessary to 250 mg/kg,

Clinical Issues

  • Little experience as orphan drug

Contra-indications

Cautions and Interactions

Side effects

  • Rare
    • Vomiting
    • Abdominal pain
    • Fever
    • Tonsillitis, inflammation of the ear, nose and throat
    • Anaemia
    • Diarrheoa
    • Headache

Special advice

Intoxication (unlikely at recommended doses) produces:

  • Tachycardia
  • Sweating
  • Bronchial secretion
  • Fever
  • Restlessness

Pharmacology

References