Morquio Syndrome Research Paper

Freak suffers from the disease known as Morquio Syndrome, which affects his metabolism. The disease presents when the body is missing or doesn't have enough of a substance needed to break down long chains of sugar molecules called glycosaminoglycans. Morquio Syndrome is a rare inherited birth defect that is estimated to occur in one of every 200,000 birth. The disease may not be visible at birth but symptoms usually begin between ages 1 and 3. Morquio syndrome is an inherited condition, which means it is passed down through families. If both parents carry a non working copy of a gene related to this condition, each of their children has a 25% (1 in 4) chance of developing the disease.

The majority of glycosaminoglycan such as KS is produced in the cartilage and therefore accumulated there, causing a direct impact on cartilage and bone development. The Skeletal system is therefore most affected but there is also involvement of the visual system, auditory system, cardiovascular system and respiratory system. Symptoms are treated as they occur. A spinal fusion may prevent permanent spinal cord injury in people whose neck bones are underdeveloped. Bone problems can lead to significant
Food and Drug Administration has approved a medicine for Morquio syndrome, called elosulfase alfa (Vimizim). It is given through a vein (IV, intravenously). Symptoms are treated as they occur. A spinal fusion may prevent permanent spinal cord injury in people whose neck bones are underdeveloped. Counseling is recommended for families who have a child with Morquio syndrome to help them understand the condition and possible treatments. Morquio syndrome is treated in many ways, because the condition can affect several body systems. The treatment a child needs depends on the severity of his symptoms. Some children might only require careful monitoring. Others may need nonsurgical or surgical treatments to address specific aspects of their