Morquio syndrome
| Morquio syndrome | |
|---|---|
| Other names | Mucopolysaccharidosis IV, MPS IV, Morquio-Brailsford syndrome, or Morquio |
| Patient with Morquio syndrome | |
| Specialty | Endocrinology |
| Complications | Skeletal abnormalities, hearing loss, pulmonary failure, heart disease |
| Usual onset | Birth; condition usually becomes apparent between ages 1 and 3 |
| Duration | Lifelong |
| Types | Type A and Type B |
| Causes | Inherited deficiency of enzymes |
| Treatment | Elosulfase alfa (Vimizim) for Type A; no approved treatment for Type B |
| Prognosis | Reduced lifespan. Usually death occurs in 20s to 30s |
| Frequency | 1 in 200,000 to 1 in 300,000 |
Morquio syndrome, also known as mucopolysaccharidosis type IV (MPS IV), is a rare metabolic disorder in which the body cannot process certain types of sugar molecules called glycosaminoglycans (AKA GAGs, or mucopolysaccharides). In Morquio syndrome, the specific GAG which builds up in the body is called keratan sulfate. This birth defect, which is autosomal recessive, is a type of lysosomal storage disorder. The buildup of GAGs in different parts of the body causes symptoms in many different organ systems.: 544 In the US, the incidence rate for Morquio syndrome is estimated at between 1 in 200,000 and 1 in 300,000 live births.