Carglumic Acid: An Emerging “Weapon” In The Treatment Of Acute Hyperammonemia
Carglumic acid, sold under brands such as Carbaglu, is used as an adjuvant therapy for the treatment of acute hyperammonemia caused by propionic acidemia (PA) or methylmalonic acidemia (MMA).
Carbaglumic acid promotes ammonia detoxification and urea production by activating carbamoyl phosphate synthase 1, thereby improving or restoring the function of the urea cycle.
What Is Hyperammonemia?
Hyperammonemia, or elevated ammonia levels, is a metabolic disorder characterized by high levels of ammonia in the blood.
Severe hyperammonemia is a dangerous disease that can lead to brain damage and death.
It may be primary or secondary. Ammonia is a nitrogen-containing substance. It is a product of protein breakdown metabolism.
It is converted into the less toxic substance urea before being excreted through urine in the kidneys.
The metabolic pathway for synthesizing urea involves a reaction that starts from mitochondria and then enters the cytosol.
This process is called the urea cycle, which consists of several enzymes acting in sequence.
Common zinc deficiency can greatly exacerbate this condition, thereby further increasing ammonia levels.
Carglumic Acid：A Breakthrough Therapy
Carbaglu Carglumic acid mainly targets the rare and challenging condition of congenital hyperammonemia.
Patients with congenital hyperammonemia are unable to metabolize ammonia normally due to the deficiency or abnormal function of certain key enzymes in their bodies, resulting in a sharp increase in ammonia concentration in the blood.
The toxic effects of ammonia can seriously damage the nervous system of patients, causing serious consequences such as intellectual disability, seizures, coma, and even death.
Previously, there were many limitations in the treatment methods for this disease, which greatly affected the quality of life and life expectancy of patients.
Clinical Efficacy And Patient Benefits
Carglumic acid has shown significant clinical efficacy in controlling acute hyperammonemia attacks and providing long-term ammonia control for UCD patients.
Carbaglumic acid is a major breakthrough for patients with NAGS deficiency, as it can improve metabolic stability, reduce the need for frequent visits, and minimize the risk of neurological damage caused by elevated ammonia levels.
Recent clinical studies have shown that when used as part of a comprehensive management strategy, Carbaglumic acid can significantly reduce blood ammonia levels and contribute to better outcomes in children’s neurological development and cognitive function.
The Future Path: New Hope For Acute Hyperammonemia Patients
The approval and widespread use of Carbaglumic acid marks an important milestone in the treatment of acute hyperammonemia.
For patients who have previously faced limited treatment options, Carbaglumic acid provides a life-changing treatment option.
However, DengYue’s efforts to increase the affordability of the medicine are critical to ensuring that more patients benefit from it. So please feel free to contact us for Carbaglu.
Information from DengYueMedicine, China Drug Import and Export Wholesaler offers this information as a guide, not a substitute for professional medical advice. Consult your doctor before any treatment.
