What is it?

Mucopolysaccharidosis type VII (MPS VII), also known as Sly Syndrome, is a rare genetic metabolic disorder.

What causes it?

People with MPS VII do not produce enough beta-glucuronidase, an enzyme that plays a key part in the breakdown off specific sugars in the body. The lack of beta-glucuronidase leads to storage of sugars within the cells, leading to progressive damage in many organs.

What are some of the common symptoms?

  • Cataracts
  • Clouding of the corneas
  • Developmental delay
  • Enlarged spleen and liver
  • Joint stiffness
  • Hearing loss
  • Heart and lung complications
  • Short stature

How many people have it?

It is estimated that fewer than 1 in 1,000,000 people have MPS VII.1

1Mucopolysaccharidosis type 7. Orpha.net rare diseases search website. https://www.orpha.net/consor/cgi-bin/Disease_Search.php?lng=EN&data_id=40&Disease_Disease_Search_diseaseGroup=Mucopolysaccharidosis-type-7&Disease_Disease_Search_diseaseType=Pat&Disease(s)/group%20of%20diseases=Mucopolysaccharidosis-type-7&title=Mucopolysaccharidosis-type-7&search=Disease_Search_Simple. Accessed August 14, 2018.

*These organizations are an incomplete listing of rare disease support organizations and are not controlled by, endorsed by, or affiliated with Ultragenyx Pharmaceutical Inc. The list is meant for informational purposes only and is not intended to replace your healthcare professional’s medical advice. Ask your doctor or nurse any questions you may have about your disease or treatment plan. If you would like to have your group added to the list, please contact patientadvocacy@ultragenyx.com.