MPS Center News

A recent publication by Matthew Hirsch, PhD, Associate Professor of Ophthalmology and Microbiology & Immunology at UNC’s Center for Molecular Medicine, advances UNC’s MPS I corneal gene therapy work. The study demonstrates reversal of corneal clouding in an MPS VI feline model and, together with existing MPS I canine data,...

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic decline. Joseph Muenzer, MD, PhD, the Director of the Muenzer MPS Research and Treatment Center and the Bryson Distinguished Professor in Pediatric Genetics at...

The 2025 MPS MasterClass brought together clinicians, researchers, families, and advocates for two days of focused education and collaboration, drawing more than 360 total views across its live Zoom sessions. Hosted by the Muenzer MPS Research & Treatment Center, the virtual program featured expert-led presentations on diagnosis, treatment advancements, clinical...

Monika Williams, MD, Assistant Professor in Pediatric Genetics & Metabolism and new MPS Center team member, has been selected for the 2025–2026 Passing the Torch cohort — a faculty development program that advances person-centered patient care and teaching through biweekly small-group learning and a capstone “passion project.” Dr. Williams focuses...

Executive Director, Dr. Kim Stephens, represented the Muenzer MPS Research & Treatment Center at the FDA Convening On the RISE: Controls in Rare Disease Clinical Trials for Small and Diminishing Populations workshop in Washington, D.C., hosted in partnership with the Duke-Robert J. Margolis, MD, Institute for Health Policy. This important meeting...

In this BioCentury op-ed, Dr. Joseph Muenzer joins other advocates in calling for a fit-for-purpose regulatory pathway for ultrarare diseases. Standard placebo-controlled clinical trials are often not feasible for these conditions, especially those affecting the brain where symptom onset and progression vary greatly between patients. These challenges can add years to...

Chapel Hill, NC – The Muenzer MPS Research & Treatment Center recently traveled to San Francisco, California, where the team led a MasterClass on Mucopolysaccharidosis (MPS) at Denali Therapeutics. The visit highlighted the importance of collaborative partnerships in advancing research, education, and care for individuals affected by MPS. During the...

Chapel Hill, NC — The team at the Muenzer MPS Research & Treatment Center joined the global rare disease community in recognizing MPS Awareness Day by proudly wearing purple—a color symbolizing strength, resilience, and unity across the MPS community. Muenzer MPS Center Team in purple for MPS Awareness Day Mucopolysaccharidoses (MPS)...

Team members from the Muenzer MPS Research & Treatment Center took part in the Raleigh Run for Rare, a 5K run and 1-mile walk hosted by the National MPS Society at Lake Crabtree County Park in Morrisville, NC. The event brought together families, advocates, and supporters to raise awareness and...

UNC Children’s has opened the Muenzer MPS Research and Treatment Center dedicated to improving the lives of patients with mucopolysaccharidoses (MPS), a group of rare inherited conditions, and advancing research with the goal of creating better therapies. UNC Children’s Opens Muenzer MPS Center in Chapel Hill

The School of Medicine’s pediatrics department will use a $10 million gift to establish a center to identify new ways to treat mucopolysaccharidoses, or MPS, a group of rare genetic disorders. The gift, from Vaughn and Nancy Bryson, who in 1960 both graduated from UNC’s School of Pharmacy, will create...