The right diagnosis made an incredible difference in my life.

Mary, a person living with NAGS Deficiency

READ MARY'S STORY

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.

Learn more about NAGS deficiency

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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.

Learn more about CARBAGLU

Indications and Usage

Carbaglu® (carglumic acid) tablets for oral suspension 200mg is a prescription drug used in all ages to help treat the following:

  • CARBAGLU is used to supplement standard of care treatment for acute high blood ammonia levels (hyperammonemia) due to the lack or shortage of NAGS enzyme activity.
  • CARBAGLU is used to maintain normal blood ammonia levels due to the lack or shortage of NAGS enzyme activity.
  • CARBAGLU is used to supplement standard of care treatment for acute high blood ammonia levels due to propionic acidemia (PA) or methylmalonic acidemia (MMA).

Important Safety Information

  • Contraindications: None.
  • NAGS deficiency: Most common side effects in ≥13% 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, hemoglobin decreased in red blood cells, and headache.
  • PA and MMA: Most common side effects in ≥5% of patients are: lower than normal white blood cells, reduced red cells in the blood, vomiting, abnormal levels of minerals in the body, decreased appetite, low blood sugar levels, lack of energy/unresponsiveness, brain disease that alters brain structure or function, and inflammation of the pancreas/increase in a type of protein made by the pancreas that helps the body digest fats.
  • 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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