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What Is Myozyme?

Jacquelyn Gilchrist
Jacquelyn Gilchrist

Myozyme® is a medication categorized as a lysosomal glycogen-specific enzyme. This means that it works by providing the patient with a specific enzyme that the patient lacks due to a genetic disorder, called Pompe disease. The medicine may benefit patients with Pompe disease, which can cause heart problems, by helping them live without the use of a ventilator. Myozyme® is not a cure for Pompe disease, so the patient must continue to take it to treat the disorder.

Prior to undergoing treatment with Myozyme®, patients may need to have a laboratory analyze their blood, muscle tissue, or skin samples. They will also need to have blood tests periodically to ensure that the drug is not resulting in adverse effects. This drug is administered by a healthcare professional via an intravenous infusion. Patients will go to the clinic about every two weeks for a dose. Each infusion takes approximately four hours to complete.


Certain precautions should be followed while undergoing treatment with Myozyme®. Before receiving a dose, patients must inform the doctor if they have recently been sick with any kind of illness. This drug may cause a serious reaction while it is being administered, or for up to three hours following an infusion. Patients should inform the doctor immediately if they experience dizziness, nausea, or a rapid or slow heartbeat. The drug may also cause problems breathing, tightness in the chest or throat, or the feeling that the patient may pass out.

Other adverse reactions during an infusion can include cold hands and blue lips, seizures, and feeling unusually hot or cold. Sweating, pale skin, or flushing may occur, as well as nervousness or restlessness. Back pain, numbness, and swelling of the throat or facial region have also been reported.

Additional serious side effects that may occur at any time during treatment may include skin ulcers, slow breathing, or a weak pulse. Rapid, slow, or uneven heartbeats may occur, as well as problems with hearing, ear pain, or a feeling of fullness in the ear. Some patients have reported chest pain, along with pain that spreads to the shoulder.

Not all side effects require immediate medical care, unless they become severe. Some of the minor side effects of Myozyme® may include stomach upset, vomiting, and diarrhea, as well as constipation, mild neck pain and a mildly sore throat. The area around the injection site may become painful, irritated, or swollen.

Before using Myozyme®, patients should disclose their other medical conditions, medications, and supplements. Women who are pregnant or breastfeeding should inform their doctors. This drug may be contraindicated for use by those with certain allergies, heart disease, or breathing disorders.

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