Donate to Morquio Syndrome

Morquio syndrome is a rare inherited disorder that primarily affects a child’s bones and spine but can also affect organs like the heart and liver. Symptoms typically appear between ages 1 and 3, including an abnormal curvature of the spine, short neck, knock knees, and hip problems.

Morquio syndrome is caused by a genetic defect that prevents the production of enzymes that break down sugar molecules called glycosaminoglycans (GAGs). Without these enzymes, GAGs can accumulate in bones, blood, tendons, and ligaments, causing damage over time.

Morquio syndrome is an autosomal recessive disorder, meaning that two copies of the mutated gene are needed—one from each parent—for symptoms to develop. Those with only one gene are "carriers" and will have no symptoms. If both parents carry the mutation, the chance of the child developing Morquio syndrome is 25%.

Morquio syndrome differs from one person to the next, with some having mild symptoms and others managing multiple severe complications. Those with mild Morquio syndrome can live near-normal life expectancies with the proper management and care. Those with severe Morquio syndrome may have a life expectancy of around 30 years.

There is no cure for Morquio syndrome but symptoms can be managed with a multidisciplinary team of providers, but to have these treatments and surgeries done are quite costly. But we want to help make it easier on every families lives giving them access to the best care in the world that we can provide.