Scientists launch 3-Year hunt for clues to heart risks in rare disease

NCT ID NCT05063435

Summary

This study aims to better understand how a group of rare genetic disorders, called mucopolysaccharidoses (MPS), affects heart and blood vessel health over time. Researchers will follow 30 patients for three years, using annual heart ultrasounds, neck artery scans, and blood tests to track changes. The goal is to gather detailed information about disease progression, not to test a new treatment.

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Contacts and locations

Locations

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

Conditions

Explore the condition pages connected to this study.