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Tracking Disease Progress to Transform Treatment

Our registry of over 400 patients with Morquio A helps us understand how the disease develops over time. We’re documenting detailed natural history data to reveal patterns that guide doctors in providing better care and helps researchers develop more effective treatments. We've published more than 50 papers and developed new treatments targeting bone health.

Revolutionizing Patient Monitoring Through Child-Friendly Tests

We’re developing gentle ways to monitor children's health. Our non-invasive, innovative approaches track breathing, bone strength, flexibility, and movement in ways that work better for kids with limited mobility. This helps us understand how well treatments are working while keeping children comfortable.

Advancing Diagnostic Precision Through Biomarker Research

We pioneer advanced methods to measure key substances (glycosaminoglycans or GAGs) that build up in Morquio A. Using innovative technology, we can precisely measure these biomarkers to diagnose the condition accurately and track how well treatments are working. This work is essential for both early detection and monitoring treatment effectiveness.

Detecting Disease Earlier to Prevent Complications

Early diagnosis can make a difference in treatment outcomes. We've developed a new screening method that can identify Morquio A and other similar conditions in newborns using just a tiny drop of blood. In our studies, we successfully identified affected infants, allowing for earlier treatment and better outcomes.

Pioneering Breakthrough Treatment Strategies

We're exploring multiple promising treatments, including stem cell transplantation, gene therapy using viral vectors, and innovative enzyme replacement approaches. We focus on developing treatments that can effectively reach and strengthen bone tissue, which is especially important for children with Morquio A.