About MPS VI

MPS VI (or Maroteaux‐Lamy syndrome) is a rare but serious genetic disease. Only about 1100 people in the world are thought to have it. People born with MPS VI tend to have a cluster of symptoms that may include:

  • Short stature
  • Coarse facial features
  • Stiff joints
  • Breathing problems
  • Difficulty walking/hip pain

A lysosomal storage disorder

The name “Maroteaux-Lamy” comes from the doctors who first described the disease in 1963. The other name of the disease, MPS VI, stands for the medical name of the disease, mucopolysaccharidosis VI.

MPS VI is a kind of disease called a “lysosomal storage disorder” or LSD. There are more than 40 known LSDs. In all of these diseases, the lack of specific enzymes causes waste to build up inside cells.

Inherited—and often hard to diagnose

MPS VI is an inherited condition. A child who gets one MPS VI gene from each parent will have the disease. There are different degrees of MPS VI. Some people have clear symptoms very early that grow worse quickly. Doctors call this “rapidly progressing” MPS VI. Some people may not show signs until later and have symptoms that get worse more slowly. This is called “slowly progressing” MPS VI. Regardless of whether a person has “rapidly” or “slowly” progressing MPS VI, the disease gets worse over time in all cases. This is why it is important to begin therapy as early as possible.

A treatment for MPS VI

NAGLAZYME® (galsulfase) replaces the natural enzyme that is missing in MPS VI. In timed tests, people who took NAGLAZYME were able to climb more stairs and walk farther than before.

In this section of Naglazyme.com, you can learn more about MPS VI, including how it affects the body and how it is diagnosed.

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View video: Dr. Chester Whitley talking about MPS VI.
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Important Safety Information

Indication

NAGLAZYME® (galsulfase) is indicated for patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy Syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity.

Important Safety Information

Severe and life-threatening allergic reactions can occur during NAGLAZYME (galsulfase) 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.

In clinical trials, most patients developed antibodies to NAGLAZYME treatment. There was no clear relationship between antibody formation and the safety or effectiveness of NAGLAZYME.

Serious and severe infusion reactions are associated with NAGLAZYME, including 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.

The most common side effects of NAGLAZYME seen in clinical trials 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.

These are not all of the possible side effects with NAGLAZYME. Talk to your doctor if you have any symptoms that bother you or that do not go away.

NAGLAZYME is a prescription medicine. Before treatment with NAGLAZYME, it is important to discuss your medical history with your doctor. Tell your doctor if you are taking any medication and if you are allergic to any medicines. Your doctor will decide if NAGLAZYME is right for you. If you have questions or would like more information about NAGLAZYME, contact your doctor.

Spinal cord damage may occur due to the natural MPS VI disease process. Signs of spinal cord injury include back pain, loss of bladder and bowel control, numbness, and paralysis. Contact your doctor immediately if you develop any of these symptoms.

To report SUSPECTED ADVERSE REACTIONS contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or go to www.fda.gov/medwatch.

Please see full Prescribing Information.