Pompe disease - FAQs

Overview | FAQs | Lysosomal storage disorders

What are the side effects of Myozyme®?
Myozyme has been known to cause cardiorespiratory failure and anaphylactic reactions. In clinical trials, the most common adverse events were pneumonia, respiratory failure, respiratory distress, catheter-related infection, respiratory syncytial virus infection, gastroenteritis, and fever. Other common reactions include diarrhea, rash, vomiting, cough, ear infection, upper respiratory tract infection, and decreased oxygen saturation. In clinical settings, the events that usually require a medical response are those related to infusion.

How is Myozyme administered?
The recommended dosage is 20 mg per kg body weight, administered intravenously every 2 weeks. The infusion should be done over about a 4-hour period. If you miss an infusion, contact your doctor or pharmacist to reschedule.

How long do I need to take Myozyme?
Your doctor determines the overall course of treatment.

How do I store Myozyme?
Myozyme should be stored in the refrigerator. Once reconstituted and diluted, protect the solution from light and administer it without delay. If immediate use isn’t possible, the solution will remain stable for up to 24 hours, as long as it is kept in the refrigerator and away from light. Do not freeze or shake it.