Alglucosidase alfa

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Alglucosidase alfa

Alglucosidase alfa (pronounced al-gloo-KOH-si-dase AL-fa) is a enzyme replacement therapy used to treat Pompe disease, a rare genetic disorder.

Etymology

The term "Alglucosidase alfa" is derived from the enzyme it replaces, alpha-glucosidase, with "alfa" being a common pharmaceutical designation for a recombinant form of a protein.

Usage

Alglucosidase alfa is used in patients with Pompe disease to replace the missing or deficient alpha-glucosidase enzyme. This enzyme is necessary for the breakdown of glycogen in the body. Without it, glycogen builds up in the body's cells, leading to the various symptoms of Pompe disease.

Administration

Alglucosidase alfa is administered via intravenous infusion under the supervision of a healthcare professional. The dosage and frequency of administration are determined by the patient's body weight and the severity of their condition.

Side Effects

Common side effects of alglucosidase alfa include allergic reactions, respiratory distress, and flu-like symptoms. Severe side effects may include anaphylaxis and severe cardiovascular events.

Related Terms

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