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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 through recruitment of new junior faculty members with support for their first three years, helping to develop more MPS-related research careers;
  • Development of funding for PhD students researching MPS, ensuring that our best and brightest in the next generation of physician researchers are supported;
  • Enhancement of educational opportunities across the UNC School of Medicine, creating learning opportunities for UNC genetic residents and fellows, pediatric residents, medical students and genetic counseling students.

Other priorities will include continuing to support the work of Dr. Muenzer’s colleague Dr. Haiyan Fu and her successful MPS gene therapy program, funding both existing MPS clinical trials and developing new trials at UNC Health, and housing MPS natural history data to inform future work.


Clinical Trials for MPS at UNC

Ongoing trials (closed to enrollment)

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Takeda (Shire): Hunter IT (intrathecal) enzyme replacement therapy

Phase I/II (HGT-HIT-046): A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase

Phase II/III (HGT-HIT-094): Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)

Takeda: TAK609 post-trial access program: Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Sangamo: Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II


Ongoing trials (open for enrollment)

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Denali: A Study of DNL310 in Pediatric Participants With Hunter Syndrome

Denali: A study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter syndrome:

Denali: A Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Patients with Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nn MPS II):


Upcoming trials

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RegenxBio: CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

JCR Pharmaceuticals: A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

Homology: Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II