Brief information about it:
Hunter syndrome is a rare genetic disorder caused by a deficiency of the enzyme iduronate-2-sulfatase (IDS). This enzyme is responsible for breaking down a complex sugar molecule called dermatan sulfate. When IDS is deficient, dermatan sulfate accumulates in the body’s cells, leading to various symptoms.
Uses:
Treatment of Hunter syndrome: Enzyme replacement therapy (ERT) is the primary treatment for Hunter syndrome. The ERT medication Aldurazyme (idursulfase) is used to replace the missing IDS enzyme.
Mechanism of action:
Enzyme replacement therapy (ERT) for Hunter syndrome works by:
- Replacing the missing enzyme: Aldurazyme provides a synthetic version of the IDS enzyme, which helps break down dermatan sulfate.
- Reducing symptoms: By reducing the buildup of dermatan sulfate, ERT can help alleviate symptoms of Hunter syndrome, such as coarse facial features, enlarged liver and spleen, intellectual disability, and joint problems.
- Preventing complications: ERT can help prevent the progression of Hunter syndrome and reduce the risk of serious complications, such as heart problems and respiratory difficulties.
Elimination half life:
The elimination half-life of Aldurazyme can vary depending on individual factors. It is generally excreted from the body within a few days.
Route of Administration:
Aldurazyme is administered intravenously, meaning it is injected directly into the bloodstream
Side effects:
Aldurazyme can cause side effects, including:
- Infections: Aldurazyme can weaken the immune system, making you more susceptible to infections.
- Allergic reactions: Allergic reactions to Aldurazyme are rare but possible.
- Infusion reactions: Some patients may experience infusion reactions, such as fever, chills, or flushing.
Dose:
The recommended dose of Aldurazyme varies depending on the severity of Hunter syndrome and individual factors. It is typically administered as a regular infusion, either every two weeks or every month.
Precautions:
· Consult a doctor: Before receiving Aldurazyme, 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 Aldurazyme.
