The right diagnosis made an incredible difference in my life.

Mary, a person living with NAGS Deficiency

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This is an actual patient story, but the patient’s name has been changed and a stock photo has been used to protect her privacy.

What is NAGS deficiency?

NAGS (N-acetylglutamate synthase) deficiency is a rare genetic disorder that blocks or limits your body’s ability to remove excess ammonia. This leads to excess ammonia building up in your blood. High blood ammonia levels may lead to brain damage or even death if symptoms are not treated.

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Treatment with CARBAGLU

Learn more about NAGS deficiency and how CARBAGLU® (carglumic acid) can help return your high ammonia levels to normal.

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Indications and Usage

Carbaglu® (carglumic acid) is a prescription drug used in all ages to help treat a rare inherited disorder called NAGS deficiency, a lack or shortage of NAGS enzyme activity, which leads to high blood ammonia levels (hyperammonemia).

  • CARBAGLU is used to supplement treatment of acute hyperammonemia due to the lack or shortage of the NAGS enzyme. Other ammonia lowering therapies such as other medications, hemodialysis and limiting protein in the diet are recommended during an acute episode.
  • CARBAGLU is used to maintain normal blood ammonia levels in patients with NAGS deficiency. During maintenance therapy, the use of other ammonia lowering therapies and limiting protein may be needed based on plasma ammonia levels.

Important Safety Information

  • Hyperammonemia: Monitor plasma ammonia level during treatment. Prolonged exposure to elevated plasma ammonia level can result in brain injury or death. Prompt use of all therapies necessary to reduce blood ammonia level is essential.
  • Most common side effects in >9% of patients are: vomiting, abdominal pain, fever, inflammation of the tonsils, reduced red cells in the blood, diarrhea, ear infection, infections, inflammation of the throat and nasal passages, protein molecules in red blood cells decreased, and headache.
  • To report SUSPECTED SIDE EFFECTS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
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