Brief information about it:
Fabry disease is a rare genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A (α-gal A). This enzyme is responsible for breaking down a fatty substance called globotriaosylceramide (GL-3). When α-gal A is deficient, GL-3 accumulates in the body’s cells, leading to various symptoms.
Uses:
Treatment of Fabry disease: Enzyme replacement therapy (ERT) is the primary treatment for Fabry disease. The ERT medication Lumizyme (agalsidase alfa) is used to replace the missing α-gal A enzyme.
Mechanism of action:
Enzyme replacement therapy (ERT) for Fabry disease works by:
- Replacing the missing enzyme: Lumizyme provides a synthetic version of the α-gal A enzyme, which helps break down GL-3.
- Reducing pain and discomfort: By reducing the buildup of GL-3, ERT can help alleviate symptoms such as pain, fatigue, and kidney problems.
- Preventing complications: ERT can help prevent the progression of Fabry disease and reduce the risk of serious complications, such as heart failure and stroke.
Elimination half life:
The elimination half-life of Lumizyme can vary depending on individual factors. It is generally excreted from the body within a few days.
Route of Administration:
Lumizyme is administered intravenously, meaning it is injected directly into the bloodstream.
Side effects:
Lumizyme can cause side effects, including:
- Infections: Lumizyme can weaken the immune system, making you more susceptible to infections.
- Allergic reactions: Allergic reactions to Lumizyme are rare but possible.
- Infusion reactions: Some patients may experience infusion reactions, such as fever, chills, or flushing.
Dose:
The recommended dose of Lumizyme varies depending on the severity of Fabry disease and individual factors. It is typically administered as a regular infusion, either every two weeks or every month.
Precautions:
· Consult a doctor: Before receiving Lumizyme, consult with a healthcare professional to ensure it is safe for you and to discuss potential risks and benefits.
· Medical history: Inform your doctor about any personal or family history of conditions such as allergies, infections, or immune system disorders.
· Infections: Avoid exposure to infections before and after receiving Lumizyme.
