Dosages
Side effects in children
Warnings & precautions in children
Contra-indications in children

Interactions
PK
Renal impairment
References

Carglumic acid

Generic name
Carglumic acid
Brand name
ATC Code
A16AA05

Pharmacokinetics in children

No information

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dose recommendation of formulary compared to licensed use (on-label versus off-label)

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Available formulations

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Dosages

Hyperammonaemia in patients with N-acetylglutamate synthase (NAGS) deficiency or organic acidaemias (isovaleric acidaemia, methylmalonic acidaemia and propionic acidaemia); CPS
  • Oral
    • 1 month up to 18 years
      • Initial dose: 100 - 250 mg/kg/day in 2 - 4 doses.
      • Maintenance dose: Titrate individually to guarantee normal plasma ammonia concentrations. Daily doses range from 10 - 100 mg/kg/day in 2 - 4 doses.
    • Term neonate
      • Initial dose: 100 - 250 mg/kg/day in 2 - 4 doses.
      • Maintenance dose: Titrate individually to guarantee normal plasma ammonia concentrations. Daily doses range from 10 - 100 mg/kg/day in 2 - 4 doses.

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Renal impaiment in children > 3 months

No information available on dose adjustment in renal impairment.

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The complete list of all undesirable drug reactions can be found in the national Summary of Product Characteristics (SmPC) – click here

Side effects

No information is present at this moment.

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The complete list of all contra-indications can be found in the national Summary of Product Characteristics (SmPC) – click here

Contra-indications

No information available on specific contra indications in children.

The complete list of all warnings and precautions can be found in the national Summary of Product Characteristics (SmPC) – click here

Warnings & precautions

No information available on specific warnings and precautions in children.

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Interactions

The complete list of all interactions can be found in the national Summary of Product Characteristics (SmPC) – click here

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References

  1. Blau, Hoffmann, Leonard and Clarke, Physicians guide to the treatment and follow-up of metabolic diseases., Springer, 2006
  2. Recordati Rare Diseases, SPC Carbaglu (EU/1/02/246/001) 09-10-2019, www.ema.europa.eu
  3. Valayannopoulos V. et al, Carglumic acid enhances rapid ammonia detoxification in classical organic acidurias with a favourable risk-benefit profile: a retrospective observational study, Orphanet Journal of rare diseases, 2016, 11, 32

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Changes

Changes