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Department of Pediatrics

Research & Clinical Trials

What We Do

Under the leadership of Dr. Muenzer, UNC has moved to the forefront of MPS discovery through more than two decades of clinical trial work and research. Dr. Muenzer’s career has included the creation of a mouse model – an invaluable research tool – for MPS II and the publication of more than 50 articles relating to the MPS disorders.

The Muenzer MPS Research and Treatment Center builds upon that existing work in several important ways:

  • Expansion of the UNC MPS basic science and translational research efforts through recruitment of new faculty members with support for their early years, prioritize junior faculty and encouraging MPS related research careers.
  • Development of funding for PhD students researching MPS, ensuring that our best and brightest in the next generation of researchers are supported;
  • Enhancement of educational opportunities across the UNC School of Medicine, creating learning opportunities for UNC residents and fellows, medical students, and genetic counseling students.

Resources

This toolkit from Global Genes with a step-by-step guide to walk families through the process of joining and participating in a clinical trial from start to finish. One of the key aims of this guide is to help families advocate for themselves and their loved ones throughout the process. It is not MPS specific but mayprovide useful information.

The National Organization for Rare Diseases (NORD) has developed a free educational series to help teach patients and families about the steps involved in developing a new drug treatment, particularly for rare diseases. The goal of this educational series is to provide families with the knowledge to empower them to effectively engage with and contribute to the drug development process.