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Carbaglu
Carglumic Acid
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About Carbaglu
Overview
Carbaglu contains carglumic acid and is used to treat high blood ammonia levels in children and adults with certain metabolic disorders. For N-acetylglutamate synthase (NAGS) deficiency, it is used alongside standard care for acute episodes and as maintenance treatment for ongoing high ammonia levels. It is also used alongside standard care for acute high ammonia levels caused by propionic acidemia (PA) or methylmalonic acidemia (MMA). It works by activating a liver enzyme that helps convert ammonia into urea, which leaves the body through urine.
What it treats
- Acute high blood ammonia levels due to N-acetylglutamate synthase (NAGS) deficiency, alongside standard care. - Chronic high blood ammonia levels due to NAGS deficiency, as maintenance treatment. - Acute high blood ammonia levels due to propionic acidemia (PA) or methylmalonic acidemia (MMA), alongside standard care. These uses apply to children and adults.
Safety information
Side effects
Common side effects reported in people with NAGS deficiency include vomiting, stomach pain, fever, tonsillitis, anemia or reduced hemoglobin, diarrhea, ear and other infections, inflammation of the nose and throat, and headache. Common side effects reported in people with PA or MMA include low levels of neutrophils (a type of white blood cell), anemia, vomiting, imbalances in blood salts, reduced appetite, low blood sugar, marked drowsiness or reduced alertness, disturbed brain function (encephalopathy), and inflammation of the pancreas or increased levels of the enzyme lipase. Serious effects that warrant contacting a doctor include low white blood cell counts, anemia, blood salt imbalances, low blood sugar, marked drowsiness or reduced alertness, encephalopathy, and inflammation of the pancreas.
When to call your doctor
If symptoms are severe or getting worse quickly, seek emergency care rather than waiting.
Before taking this medicine
The FDA label lists no contraindications for Carbaglu. The supplied section does not specify conditions requiring caution.
Using Carbaglu
Dosing
Dosing is set by your prescriber. Each health care professional may prescribe medication differently according to a patient's medical history and condition, so follow the directions on your prescription label and speak to your doctor or pharmacist before changing anything.
Storage
Store below 25°C in a dry place, in the original pack, away from direct light and moisture. Do not keep it in a bathroom. Keep all medicines out of the sight and reach of children.
Clinical information on this page is summarised from the US FDA label CARBAGLU (CARGLUMIC ACID) TABLET, FOR SUSPENSION [RECORDATI RARE DISEASES] (DailyMed, retrieved 19 September 2026).
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