Mucopolysaccharidosis (MPS) is a rare disease in which the body is missing or does not have enough of an enzyme needed to break down long chains of sugar molecules. These chains of molecules are called glycosaminoglycans (formerly called mucopolysaccharides). As a result, the molecules build up in different parts of the body and cause various health problems.
We are honored to support MPS Awareness Day, happening on May 15th. MPS Awareness Day helps to drive awareness of MPS and hopefully throughout the year with providers, patients, caregivers, and more.
If you’re interested in learning more about MPS, here are eight important facts and statistics that may be helpful.
1. There are 9 subdivisions of MPS that have been identified:
- MPS 1 H/S (Hurler/Scheie syndrome)
- MPS I H (Hurler disease)
- MPS II-(Hunter syndrome)
- MPS III A, B, C, and D (Sanfillipo syndrome)
- MPS I S (Scheie syndrome)
- MPS IV A and B (Morquio syndrome)
- MPS IX (hyaluronidase deficiency)
- MPS VII (Sly syndrome)
- MPS VI (Maroteaux-Lamy syndrome)
(rarediseases.org)
2. The prevalence of all subdivisions of MPS is estimated to be one in 25,000 births. However, because MPS, especially the milder forms of the disease, often go unrecognized, the disease is often under-diagnosed or misdiagnosed, making it difficult to determine its true frequency in the general population. (rarediseases.org)
3. Individuals with MPS disorders share many similar symptoms such as multiple organ involvement, distinctive facial features, and abnormalities of the skeleton, especially joint problems. (rarediseases.org)
(www.ninds.nih.gov and orsini.com)
4. Prevalence of MPS I is estimated at 1/100,000, with Hurler disease accounting for 57% of cases, Hurler-Scheie syndrome accounting for 23% of cases, and Scheie syndrome accounting for 20% of cases. https://www.orpha.net/
5. MPS II typically affects only males, with symptoms becoming apparent at approximately 2-4 years of age. https://rarediseases.org/
6.MPS III and MPS IV are inherited disorders. If both parents carry a nonworking copy of a gene related to this condition, each of their children has a 25% (1 in 4) chance of developing the disease. https://medlineplus.gov/
7. Currently, there is no cure for MPS. Medical care and medications are directed at treating systemic effects of the condition. Medications include:
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- ALDURAZYME® (laronidase)
- ELAPRASE® (idursulfase)
- MEPSEVII® (vestronidase alfa-vjbk)
- NAGLAZYME® (galsulfase)
- VIMIZIM® (elosulfase alfa)
8. There are a number of emerging treatments and therapies for MPS currently in development. You can click here to learn more: https://mpssociety.org/learn/treatments/emerging-treatments/
Orsini Specialty Pharmacy prescribes four of these therapies. See how we can support MPS patients.