MPS disorders are a group of rare, genetic, inheritable, metabolic diseases, which form part of the lysosomal storage diseases (LSDs)11–14

  • They affect males and females equally (with the exception of MPS II, which mainly affects males) and can be as common as ~1 in 25,000 live births (with prevalence varying by region and ethnic background)13,15
  • MPS disorders are caused by a deficiency in enzymes responsible for degrading glycosaminoglycans (GAGs) [see table below]. As a result, GAGs accumulate in the lysosomes of many of the body’s cells, causing progressive damage throughout the body11–14
  • Multiple organs and systems can be involved, including the cardiovascular, skeletal, respiratory and neurologic systems13
  • Having an MPS disorder can lead to organ failure, cognitive impairment and reduced life expectancy13-14

To date, 11 different enzymatic deficiencies are known to cause seven MPS disorders:12–14

For more information about MPS, please visit www.MPSReference.com.