MPS Center News

GSD Type I Presentation Highlights from Dr. Monika Williams The Muenzer MPS Research & Treatment Center recently hosted a journal club presentation by Dr. Monika Williams, Assistant Professor of Pediatrics in Genetics & Metabolism at UNC, highlighting key insights into Glycogen Storage Disease Type I (GSD I). ⬇ Download Presentation...

A recent story from CBS 17 Raleigh, North Carolina, highlights the journey of Dr. Kim Stephens and her family’s experience living with Hunter syndrome (MPS II). The article also features ongoing research efforts at the UNC School of Medicine focused on developing new treatment approaches for rare genetic disorders. In...

Members of the Muenzer MPS Research & Treatment Center team recently participated in a Rare Disease awareness event at UNC Health, engaging with healthcare professionals, patients, and hospital visitors to help increase understanding of rare diseases. During the event, the team shared educational resources and spoke with members of the...

During Rare Disease Week, Dr. Kim Stephens was on Capitol Hill in Washington, D.C., representing the UNC Muenzer MPS Research & Treatment Center and the broader rare disease community. Advocacy is an important part of advancing research, improving access to care, and ensuring that the needs of individuals and families...

On February 12, 2026, at UNC Department of Pediatrics Grand Rounds, Liz Jalazo, MD, Assistant Professor of Pediatrics in Genetics & Metabolism, presented on the growing impact of gene therapy in neuromuscular disease care. Her presentation explored how advancements in spinal muscular atrophy (SMA) treatment are helping shape future approaches...

The Muenzer MPS Research & Treatment Center participated in the 2026 WORLD Symposium held February 2–6, 2026, in San Diego, California, joining international leaders in lysosomal disease research and clinical care. Center faculty and staff contributed multiple presentations and scientific posters addressing key topics across several MPS disorders: Dr. Elizabeth...

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...