UNC Muenzer MPS Center

10/14/2026

More than 1,000 views and counting!

A publication co-authored by Dr. Kim Stephens, Executive Director of the Muenzer MPS Research & Treatment Center, has surpassed 1,000 views since its publication in October 2025.

The study, “The spectrum of communication abilities in children with twelve rare neurodevelopmental disorders: A qualitative study with caregivers,” explores communication abilities among children with 12 rare neurodevelopmental disorders, including MPS II (Hunter syndrome), through the experiences and perspectives of caregivers.

This milestone highlights the growing reach of research that brings caregiver perspectives into focus and helps advance our understanding of the communication needs of children living with rare diseases.

Read the publication: https://doi.org/10.1111/jcpp.70063

10/13/2026

Kristin Clinard - UNC MPS Center, Genetic Counselor at the Muenzer MPS Research & Treatment Center, presented today at the APHL Newborn Screening Symposium in New Orleans, Louisiana.

Her presentation, “Addressing Uncertainty in Newborn Screening Follow-up for Mucopolysaccharidosis I and II Through Risk-stratified Clinical Frameworks and Disease Monitoring Guidelines,” highlighted a risk-stratified framework for assessing MPS disease risk and guiding clinical follow-up across the full spectrum of abnormal newborn screening outcomes.

Designed to support healthcare providers, including general pediatricians, the proposed framework balances the benefits of early detection with the importance of avoiding unnecessary medical interventions, while recognizing the experiences and wellbeing of families navigating screen-positive results.

10/06/2026

Sarah Bennick, MSN, CPNP-PC, and Lindsay Torrice, MSN, CPNP-PC, of the Muenzer MPS Research & Treatment Center presented at the NC NAPNAP Conference in Raleigh, North Carolina. The one-day conference, hosted by the North Carolina chapter of NAPNAP, brought together pediatric nurse practitioners from across the state.

Their presentation, “Managing MPS I & II after NBS: A Primary Care Framework for Surveillance and Risk Assessment,” focused on helping pediatric providers navigate the care, surveillance, and risk assessment of children with MPS I and MPS II following newborn screening.

10/02/2026

Italfarmaco and JCR Pharmaceuticals have announced an agreement to develop and potentially commercialize JR-141 (pabinafusp alfa), an investigational blood-brain barrier-penetrating enzyme replacement therapy for Hunter syndrome (MPS II), in the United States, Europe, and Latin America. JR-141 is currently being evaluated in a global Phase III clinical trial.

Learn more:
https://www.businesswire.com/news/home/20261001541148/en/Italfarmaco-and-JCR-Pharmaceuticals-Enter-into-Exclusive-License-Agreement-for-the-Development-and-Potential-Commercialization-of-Novel-Investigational-Therapy-for-Hunter-Syndrome-in-the-United-States-Europe-and-Latin-America

09/30/2026

The story of the Muenzer MPS Research & Treatment Center is rooted in a shared commitment to improving the lives of individuals and families affected by MPS.

A transformational $10 million gift from UNC alumni Vaughn and Nancy Bryson helped make the Center possible and continues to support our mission to advance MPS research, treatment and comprehensive care.

Read more about the story behind the establishment of the Center: https://alumni.unc.edu/news/10-million-gift-to-establish-center-to-research-genetic-disorder/

09/24/2026

Dr. Elizabeth Jalazo, Associate Director of the Muenzer MPS Center, presented at ADVANCE 2026, Sanfilippo Community Conference, hosted by Cure Sanfilippo Foundation , on “Immune Responses in Sanfilippo Enzyme Replacement.”

During the presentation, Dr. Jalazo compared immune responses to enzyme replacement therapy across different MPS types, using what has been learned from existing ERTs to inform our understanding of immune responses to emerging therapies for Sanfilippo syndrome.

The discussion highlighted important considerations as researchers continue working to better understand and improve therapeutic approaches for individuals with Sanfilippo syndrome.

09/21/2026

Dr. Elizabeth Jalazo, Associate Director of the Muenzer MPS Research & Treatment Center, will be presenting at ADVANCE 2026 on immunogenicity in enzyme restorative therapies.

The free virtual Cure Sanfilippo Foundation community conference takes place September 22–23 and will feature research, clinical trial, and treatment updates.

Sanfilippo is changing fast. New research. New ideas. New possibilities. ADVANCE 2026 is your chance to get caught up on what’s happening and focus in on the topics you want to know more about.

Here are some highlights of what’s on the agenda for Sept. 22 & 23:
👉 Clinical Trial & Biotech Updates — Hear directly from companies and researchers developing and testing potential treatments for Sanfilippo.

👉 Immunogenicity & Enzyme Replacement Therapy — A deep dive into what are learning from other lysosomal diseases and how those lessons may apply to Sanfilippo.

👉Communication in Sanfilippo — Strategies and tools that can help families and care teams better understand and communicate with children as communication abilities changes.

From evolving treatment approaches to new research directions and practical insights for families, ADVANCE brings a wide range of Sanfilippo topics together in one virtual conference.

And because we believe these conversations should be accessible to everyone who wants to be part of them, registration is free.

👉 Register: https://pro.gofundme.com/live/register/advance-2026-sanfilippo-community-conference/8bf1c999-f506-40a1-806e-e84f94ad6bb7

Two days. A lot to explore. Come hear what’s next.

09/18/2026

Dr. Joseph Muenzer recently presented at the IX Latin American Course on Lysosomal Storage Diseases (LACLSD) in Antigua, Guatemala.

During the conference, Dr. Muenzer shared his expertise in two presentations focused on MPS:
🔹 Clinical Aspects of MPS and Related Diseases
🔹 Treatment for MPS and Related Diseases

LACLSD brings together healthcare professionals and experts in lysosomal storage diseases from across Latin America and around the world to advance education, collaboration, and care for individuals and families affected by these rare diseases.

09/17/2026

💙 Hope takes all of us.

The Muenzer MPS Research & Treatment Center is dedicated to treating individuals with mucopolysaccharidosis (MPS) and advancing research to improve the lives of those affected by MPS.

Led by world-renowned MPS expert Joseph Muenzer, MD, PhD, the Center is 100% funded through philanthropic support.

In addition to advancing MPS research and treatment, the Center provides a consultation program where patients and families can receive guidance from clinicians with extensive experience in MPS care.

Your generosity helps make this work possible and supports our continued commitment to individuals and families throughout the MPS community. 💙

📞 (919) 228-2432
✉️ [email protected]

💙 Support the Muenzer MPS Center:
go.unc.edu/MPSCenterDonate