Treatment FAQ
What is NAGLAZYME® (galsulfase)?
NAGLAZYME is a kind of treatment called Enzyme Replacement Therapy, or ERT.3 NAGLAZYME is a highly purified protein that is identical to the natural enzyme that is missing in MPS VI.1 NAGLAZYME reduces the GAG build-up that leads to MPS VI symptoms.1 In clinical studies, NAGLAZYME has been shown to help with endurance. In timed tests, people who took NAGLAZYME were able to climb more stairs and walk father than before.1
What is NAGLAZYME used for?
NAGLAZYME is a treatment for people with MPS VI.1 NAGLAZYME provides the enzyme needed to break down and get rid of the excess GAG that causes symptoms of MPS VI.1 Learn More
Could NAGLAZYME be right for me?
All patients with MPS VI can benefit from NAGLAZYME.7 Your doctor can tell you whether NAGLAZYME may be right for you. Learn More
What does the doctor need to know about me before I start treatment with NAGLAZYME?
Your doctor will need to do a physical exam. He or she may also need to know:
- If you are pregnant, may become pregnant, or are breast-feeding
- Have medical problems such as trouble breathing, even if these are not related to MPS VI
- Have allergies, even if they are not related to MPS VI
- Take other medications (prescription or over-the-counter) or supplements or vitamins
How do I take NAGLAZYME?
NAGLAZYME is given by infusion. That means that once a week, you will go to a hospital or infusion center. At the center, a doctor or nurse will give you NAGLAZYME through an intravenous (IV) line. The IV gets the enzyme right into your bloodstream. The blood carries it to your body’s cells and organs to break down GAG. Learn More
How long does an infusion take?
The infusion itself takes about 4 hours. Learn More
Will it hurt?
Starting the IV can cause some pain or discomfort. Sometimes people have reactions to the IV, such as headache or nausea. If you have any problems with your infusion, talk to your doctor or nurse. They may be able to help you feel better. Learn More
What if I’m scared of needles?
Talk with your doctor. You might be able to get a numbing cream so the needle won’t hurt. Most people get over this fear after getting their first or second infusion.
Afraid of needles?
- View and download this helpful brochure
(PDF 1.1MB)
- Also, focus on the fact that the needle is delivering the enzyme you need to be at your best. More
Also, focus on the fact that the needle is delivering the enzyme you need to be your best. Learn More
How will I feel during infusions?
Some patients feel fine and use the time to read or do quiet activities. Other patients may feel sleepy or tired. This is often due to the pre-medications. If you find you are sleepy and would rather not be, talk with your doctor. You may have other pre-medication options.
It is possible you may have a side effect from NAGLAZYME. If you don’t feel well for any reason during an infusion, let the nurse know. Learn More
What are possible side effects of NAGLAZYME?
With all medications, there is a chance of side effects. This is true of NAGLAZYME too.
Severe and life-threatening allergic reactions can occur during NAGLAZYME infusions and up to 24 hours after infusion. Typical signs of an allergic reaction include shock, difficulty breathing, wheezing, swelling of the throat, and low blood pressure. If a severe allergic reaction occurs during infusion, the infusion should be stopped immediately and you should receive medical attention. Contact your doctor or get medical help right away if you develop any severe symptoms after infusion.
Because NAGLAZYME is given by infusion, there are reactions that may happen as a result of the infusion. In clinical trials, some people experienced serious and severe infusion reactions like hives, chest pain, rash, abdominal pain, difficulty breathing, swelling, fever, and eye irritation. You should receive medication such as antihistamines before NAGLAZYME infusions to reduce the risk of infusion reactions.
If an infusion reaction occurs, the infusion should be slowed or stopped and you may be given additional medication. Most of the time, these problems went away when the infusion was stopped or slowed down. In some cases, patients were given another medicine, such as an antihistamine or pain reliever, to help.1
In clinical studies, the most common side effects of NAGLAZYME were rash, pain, hives, fever, itching, chills, headache, nausea, vomiting, abdominal pain and difficulty breathing. The most common side effects requiring medical attention are infusion-related effects.1
How long will I have to take NAGLAZYME?
MPS VI is a lifelong disease, so NAGLAZYME is a lifelong treatment.
What if I miss a treatment?
Talk with your doctor or infusion center about scheduling your next dose. Learn More
Can I take NAGLAZYME with other medications?
Always tell your doctor about other medications before you start a new treatment. Your doctor needs to know about any drugs you are taking now, as well as anything you might have stopped taking recently.
Tell your doctor about all medicines, both prescription and over-the-counter. Also tell the doctor about vitamins or other dietary supplements you take.
What changes can I expect from NAGLAZYME?
NAGLAZYME provides the enzyme the body needs to break down and get rid of GAG. In timed tests, people who took NAGLAZYME were able to climb more stairs and walk farther than before. Learn More
Will insurance cover me?
NAGLAZYME is an important treatment for all people with MPS VI. The experts at BioMarin Patient and Physician Support (BPPS) will help you with access to insurance coverage. All you have to do is call them. Learn More
Are there support programs and services to help me?
The makers of NAGLAZYME, BioMarin, have a program to help all MPS VI patients get the treatment they need. This program is called the BioMarin Patient and Physician Support program (BPPS).
When you call, you’ll have your own helpful BPPS advocate—one person you talk with every time you call. Your BPPS advocate becomes familiar with you and your case, and will help you:
- Navigate the health care system to make sure you can get the NAGLAZYME you need
- Understand NAGLAZYME treatment
- Find an infusion center near you
- Manage any problems over time, so you can keep getting treatment
You can contact BPPS at 866-906-6100, or by e-mailing bpps@bmrn.com.
Learn More