Search

Rapidly and slowly progressing MPS VI

Rapidly progressing MPS VI

Some people with MPS VI show signs early and clearly. Babies with this rapidly progressing MPS VI may not grow well. Other symptoms or medical issues may quickly emerge, including1-5:

  • Coarse facial features
  • Enlarged tongue
  • Vision problems
  • Breathing problems
  • Disease of the heart valves
  • Stiff joints

With these obvious symptoms, patients with rapidly progressing MPS VI are often diagnosed early.

Symptoms may be recognized early in life for rapidly progressing MPS VI or later in adolescence for slowly progressing MPS VI. All patients with MPS VI eventually experience significant disability and may experience a shortened life span.2

Slowly progressing MPS VI

Other people with MPS VI may have symptoms that are slower to develop. At first, some of their symptoms, like constant ear infections, may just seem like common childhood problems. On the other hand, a problem that is unusual in children, like stiff joints, may be seen as something that affects only the bones. For these “slowly progressing” patients, it can take the doctor a while to see that different symptoms, affecting many parts of the body, are all really due to MPS VI.5,6

Whether rapidly or slowly progressing, MPS VI requires treatment

There is no hard line between rapidly and slowly progressing MPS VI. There are degrees in between. Symptoms can be very different from patient to patient. Even though the symptoms are not initially clear in people with slowly progressing MPS VI, GAGs are still building up in their cells. It is still causing problems in their organs. Over time, people with slowly progressing MPS VI will notice more and more symptoms and the condition will progress. The symptoms of MPS VI need both symptom-based care and treatment with enzyme replacement therapy (ERT). All MPS VI patients are candidates for ERT treatment with NAGLAZYME® (galsulfase). NAGLAZYME provides the enzyme the body needs to break down and get rid of GAGs.2,6,7

References:

  1. Swiedler SJ, Beck M, Bajbouj M, et al. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Am J Med Genet A. 2005;134A(2):144-151.
  2. Akyol MU, Alden TD, Amartino H, et al. Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance. Orphanet J Rare Dis. 2019;14:118.
  3. Wilcox WR. Lysosomal storage disorders: the need for better pediatric recognition and comprehensive care. J Pediatr. 2004;144(suppl 5):S3-S14.
  4. Clarke L, Ellaway C, Foster H. Understanding the early presentation of mucopolysaccharidosis disorders: results of a systematic literature review and physician survey. J Inborn Errors Metab Screen. 2018;6:1-12.
  5. National MPS Society. Maroteaux-Lamy syndrome. A guide to understanding MPS VI [booklet]. Accessed March 24, 2026. https://mpssociety.org/wp-content/uploads/2025/03/MPS_VI_Final_Booklet.pdf
  6. D’Avanzo F, Zanetti A, De Filippis C, Tomanin R. Mucopolysaccharidosis type VI: an updated overview of the disease. Int J Mol Sci. 2021;22(24):13456.
  7. NAGLAZYME [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.

IMPORTANT SAFETY INFORMATION

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. These reactions have occurred during and up to 24 hours after completion of the NAGLAZYME infusion. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions, including anaphylaxis.

Initiate NAGLAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue NAGLAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. In patients who have experienced anaphylaxis or other severe allergic reactions during infusion with NAGLAZYME, caution should be exercised upon rechallenge. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.

What is the most important information I should know about NAGLAZYME?
Severe and life-threatening allergic reactions, including anaphylaxis, can occur during NAGLAZYME infusions and up to 24 hours after infusion. These reactions can occur in people receiving NAGLAZYME for the first time or in people who have previously received NAGLAZYME without having an allergic reaction.

Your doctor will tell you about the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and when to seek immediate medical care. Signs of anaphylaxis can include cough, rash, throat tightness, hives, flushing, changes in skin color, low blood pressure, shortness of breath, chest pain, and gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting. If a severe allergic reaction (e.g., anaphylaxis) 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.

What are the most common side effects of NAGLAZYME?
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.

What else should I know about NAGLAZYME?
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.

Call your doctor for medical advice about side effects. You may report side effects to BioMarin at 1-866-906-6100 and the FDA by visiting www.fda.gov/medwatch or calling 1-800-FDA-1088.

Please see full accompanying Prescribing Information, with important warning for risk of anaphylaxis or visit www.Naglazyme.com.  

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. These reactions have occurred during and up to 24 hours after completion of the NAGLAZYME infusion. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Administration of NAGLAZYME should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions, including anaphylaxis.

Initiate NAGLAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue NAGLAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. In patients who have experienced anaphylaxis or other severe allergic reactions during infusion with NAGLAZYME, caution should be exercised upon rechallenge. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.

INDICATION

NAGLAZYME® (galsulfase) is indicated for patients with mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stairclimbing capacity.