Trusted Resources: Education
Scientific literature and patient education texts
A Guide to Understanding MPS IV (Morquio Syndrome)
source: National MPS Society
year: 2021
summary/abstract:Mucopolysaccharidosis IV (MPS IV) is a rare genetic disorder characterized by short stature and severe bone disease. It is also known as Morquio syndrome, named after Dr. Morquio, a pediatrician in Montevideo, Uruguay. In 1929, Dr. Morquio described a family of four children affected by this condition. The same year, Dr. Brailsford from Birmingham, England, also described the same characteristics. Consequently, it is sometimes known as Morquio-Brailsford syndrome.
What Causes MPS IV?
In healthy individuals, GAGs are used in the building of bones, cartilage, skin, tendons, and many other tissues in the body. For instance, the slippery synovial fluid that lubricates your joints contains GAGs, as does the rubbery cartilage in your joints. All tissues have some of this substance as a normal part of their structure. As more GAGs are produced, older GAGs get broken down; this is the normal cycle of events that maintains a healthy balance in the body. However, when this cycle does not function properly and GAGs are not broken down, they accumulate within the cells. This malfunction results in progressive and sometimes permanent cellular damage, affecting the individual’s physical abilities, proper functioning of organs and systems, appearance, and, cognitive development.
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