Naglazyme: Used for Sanfilippo syndrome type A.
Brief information about it:
Naglazyme is an enzyme replacement therapy (ERT) used to treat Sanfilippo syndrome type A, a rare genetic disorder that causes the buildup of a fatty substance called heparan sulfate in the body’s cells.
Uses:
Sanfilippo syndrome type A: Naglazyme is used to replace the missing or deficient enzyme, N-sulfatase, in patients with Sanfilippo syndrome type A. This helps break down heparan sulfate, reducing the buildup of this fatty substance in the body’s cells.
Mechanism of action:
Naglazyme is a synthetic enzyme that mimics the natural N-sulfatase enzyme. By replacing the missing or deficient enzyme, Naglazyme helps break down heparan sulfate, reducing the buildup of this fatty substance in the body’s cells. This can alleviate the symptoms of Sanfilippo syndrome, such as intellectual disability, delayed development, and aggressive behavior.
Elimination half life:
The elimination half-life of Naglazyme can vary depending on individual factors. It is generally excreted from the body within a few days.
Route of Administration:
Naglazyme is administered intravenously, meaning it is injected directly into the bloodstream.
Side effects:
· Infections: Naglazyme can weaken the immune system, making you more susceptible to infections.
· Allergic reactions: Allergic reactions to Naglazyme are rare but possible.
· Infusion reactions: Some patients may experience infusion reactions, such as fever, chills, or flushing.
Dose:
The recommended dose of Naglazyme varies depending on the severity of Sanfilippo syndrome type A and individual factors. It is typically administered as a regular infusion, either every two weeks or every month.
Precautions:
· Consult a doctor: Before receiving Naglazyme, 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 Naglazyme.
