Also known as Sly syndrome, MPS 7
What is it?
Mucopolysaccharidosis type VII (MPS VII) 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 Common Symptoms?1, 2, 3
- 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
- Mucopolysaccharidosis type 7. Orpha.net rare diseases search website. https://bit.ly/3fYTG6S. Accessed August 14, 2018.
- National Organization for Rare Disorders. Mucopolysaccharidosis Type VII. NORD Website. Published 2017. Accessed October 31, 2021. https://rarediseases.org/rare-diseases/glycogen-storage-disease-type-i/
- Montaño AM, Lock-Hock N, Steiner RD, et al. Clinical course of sly syndrome (mucopolysaccharidosis type VII). J Med Genet. 2016;53(6):403-418.
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Participate in MPS VII Research
To talk to someone and get more information about Ultragenyx clinical trials and research, email [email protected].
Disease Monitoring Program
Research plays a critical part in advancing science and treatments. Volunteers who participate in this research are essential. Future therapies would not be possible without them. Participating in clinical trials, interviews, observational studies, and disease monitoring programs help to drive research, treatment development, and disease education. It is also a way to advocate for yourself and your community.
Upcoming Community Events
Participate in Events Near YouMPS VII Patient Advocacy Groups*
*These organizations are an incomplete listing of rare disease advocacy groups 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 [email protected].
Rare Experiences
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Cynthia
Mucopolysaccharidosis Type VII (MPS VII)“We would like Cynthia to know that she is not the only one with a rare disease”
Matthew
Mucopolysaccharidosis Type VII (MPS VII)“He has his own way of communicating with people, including his own language, which his family and very close friends have learned”
