Contents
Definitions
Morquio has multiple names referring to the same disability.
- Morquio Syndrome
- Morquio-Brailsford syndrome
- Morquio
- Mucopolysaccharidosis IV (MPS IV)
- MPS IV
- MPS IV A
- MPS IV B
NIH US National Library of Medicine MedlinePlus defines Morquio as: Mucopolysaccharidosis type IV (MPS IV) 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.
The condition belongs to a group of diseases called mucopolysaccharidoses (MPSs). MPS IV is also known as Morquio syndrome.
Causes
MPS IV is an inherited disorder. This means it is passed down through families. 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. This is called an autosomal recessive trait.
There are two forms of MPS IV: type A and type B.
- Type A is caused by a defect in the GALNS gene. People with type A do not have an enzyme called N-acetylgalactosamine-6-sulfatase.
- Type B is caused by a defect in the GLB1 gene. People with type B do not produce enough of an enzyme called beta-galactosidase.
The body needs these enzymes to break down long strands of sugar molecules called keratan sulfate. In both types, abnormally large amounts of glycosaminoglycans build up in the body. This can damage organs.
Go to their website to read more.
Resources
Other websites that describe what Morquio is:
- NORD National Organization for Rare Disorder
- NIH U.S. National Library of Medicine. Genetics Home Reference:
- National MPS Society
- BioMarin Pharmaceutical Inc. Morquiosity
See our Resources page for more!
Videos
These are various videos found on YouTube.
Achondroplasia and Morquio Syndrome Update:
Global HELP and COTI presents Dr. William Mackenzie, Department Director of Nemours DuPont Hospital for Children Jefferson Medical College. This video can be divided into two different parts. In the first segment, Dr. Mackenzie talks about the genetic basis of achondroplasia and physical abnormalities that often accompany the disease. The efficacy of molecular treatments for achondroplasia are evaluated through animal studies. The second segment of the video discusses the indications and characteristics of Morquio syndrome.
More videos to come.
