CBS 17 Feature
The UNC Muenzer MPS Research & Treatment Center continues to play a critical role in advancing treatments for rare diseases, contributing to research behind a newly approved therapy for Hunter syndrome (MPS II).
A recent CBS 17 story highlights the impact of this work, including a young patient from Hillsborough who participated in a clinical trial at UNC. The trial, led by Dr. Joseph Muenzer, helped support the development of a treatment designed to address both the neurological and physical symptoms of the disease.
Hunter syndrome is a rare genetic disorder that primarily affects boys and can lead to progressive damage across multiple organ systems, including the brain. Historically, available therapies have not effectively addressed the neurological aspects of the disease.
This newly approved treatment represents a significant advancement, with the potential to slow disease progression and improve quality of life for patients.
“We have some of the early enrollees, two brothers who are actually living in North Carolina. The youngest was treated under two years of age. Now, six years later, if I showed him to you, you would not recognize him as having Hunter syndrome. He’s basically developing normally.”
— Joseph Muenzer, MD, PhD
“I knew it was good. It worked. I was amazed by all the kids running around.”
— Kim Stephens, DBA
Research conducted at UNC continues to drive meaningful progress in rare disease care and treatment.
Watch the CBS 17 Feature
You can watch the full CBS 17 story below.