UNC Muenzer MPS Center

09/01/2026

Muenzer MPS Center at SSIEM 2026 🌎

Dr. Joseph Muenzer and Dr. Elizabeth Jalazo recently contributed to the scientific program at SSIEM 2026 in Helsinki, Finland, sharing research focused on emerging therapies for MPS.

Dr. Muenzer presented on tividenofusp alfa for MPS II (Hunter syndrome), while Dr. Jalazo contributed research focused on MPS IIIA (Sanfilippo syndrome type A).

The international symposium brought together researchers and clinicians from around the world to share advances in inherited metabolic disease research and care.

08/26/2026

📢 More Good News for the MPS IIIB Community!

NeuroGT has announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for NGT-104, an investigational AAV9 gene replacement therapy being developed for MPS IIIB (Sanfilippo syndrome type B).

The FDA clearance allows NeuroGT to move forward with a Phase I/II clinical trial, which is expected to begin enrolling patients in January 2027.

NGT-104 is designed as a one-time intravenous gene therapy intended to deliver a functional copy of the NAGLU gene. There are currently no FDA-approved treatments for MPS IIIB.

It is encouraging to see continued progress and multiple potential treatment approaches moving forward for the MPS IIIB community.

🔗 Read the full NeuroGT press release in the comments below.

08/26/2026

📢 MPS IIIB Research & Regulatory Update

Spruce Biosciences has announced the completion of two positive pre-BLA meetings with the U.S. Food and Drug Administration (FDA) regarding tralesinidase alfa enzyme replacement therapy (TA-ERT) for MPS IIIB (Sanfilippo syndrome type B).

According to Spruce Biosciences, the company and FDA aligned on key aspects of the planned Biologics License Application (BLA), and Spruce Biosciences remains on track to submit the BLA in Q4 2026. The company continues to pursue an accelerated approval pathway for TA-ERT.

TA-ERT is an investigational enzyme replacement therapy being developed for MPS IIIB, a condition for which there are currently no FDA-approved therapies.

🔗 The full Spruce Biosciences press release is linked in the comments below.

08/24/2026

Important Update: RGX-121 Gene Therapy Program for MPS II

Today, REGENXBIO announced that the FDA has placed its RGX-121 gene therapy program on clinical hold after enhanced monitoring identified small nodular or cystic findings along the spine in five patients. These patients remain asymptomatic, and the findings are believed to be benign. REGENXBIO is working with independent researchers to determine whether this reflects a natural, MPS-related process rather than a treatment effect.

REGENXBIO will continue monitoring all enrolled patients and does not expect to resubmit in the near term. The National MPS Society and Project Alive will keep the community informed of updates.

Please find the link to the full text of the announcements and community letter in the comments.

08/18/2026

💙 Supporting MPS Patients & Families

Living with MPS (Mucopolysaccharidosis) can bring a lot of questions — whether your family is navigating a new diagnosis, exploring treatment options, looking for specialized care, or simply trying to better understand what comes next.

At the Muenzer MPS Research & Treatment Center, patients and families are at the heart of what we do. Our multidisciplinary team is here to provide education, resources, and guidance to help families feel informed and supported throughout their MPS journey.

We encourage patients and families to connect with our team, ask questions, and learn more about the resources and specialized care available through the Muenzer MPS Center.

💻 Patient Connect Form:
https://unc.az1.qualtrics.com/jfe/form/SV_efkRtvbohN1EDAO

📧 Email: [email protected]
📞 Phone: 919-228-2432

No matter where you are in your MPS journey, our team is here to help you and your family along the way. 💙

08/12/2026

🧬 New Publication

A new open-access publication, "Mucopolysaccharidosis Type II Screening, Diagnosis, and Management: A Literature Review and Practical Recommendations for Newborn Screening Programs and Health Care Providers to Support Families and Improve Outcomes," provides a comprehensive review of MPS II (Hunter syndrome). The publication examines newborn screening, diagnosis, clinical management, and practical recommendations to help newborn screening programs and healthcare providers support affected children and their families while improving outcomes.

Read the full publication by visiting the link below:
https://www.mdpi.com/4028378

08/12/2026

The Cure Sanfilippo Foundation and the National MPS Society have announced a combined $5.5 million strategic investment in Spruce Biosciences to help expand access to investigational Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) for children with MPS IIIB (Sanfilippo syndrome type B ) through an Expanded Access Program.

Read the full announcement below to learn more.
https://investors.sprucebio.com/news-releases/news-release-details/cure-sanfilippo-foundation-and-national-mps-society-make-55?fbclid=IwY2xjawTpXxpwZG9mAWV4dG4DYWVtAjEwAGJyaWQRMVNRWHRxNjlLd2FlcVZxeVZzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEes-heDsMXSxkS_pt7634c3acJnKjiTJEKro77AjbWiNFsuRGf5gN0vIsSAT8_aem_0aDCwOLM9IT_FK8JA0EogA

08/05/2026

08/03/2026

Kristin Clinard - UNC MPS Center and Leigh Anne had a blast taking over our social media for the weekend — thanks for following along!

For Dr. Muenzer and all our providers at the UNC Muenzer MPS Center, the National MPS Society family meetings aren't just another event on the calendar — they're one of the most meaningful parts of the work we do and why we do it! Because behind every number and every diagnosis is a family!!



UNC School of Medicine UNC Health UNC Children's - North Carolina Children's Hospital

08/02/2026

Our team has had the best time seeing familiar faces and meeting new ones at the 2026 National MPS Society family meeting in Columbus, OH 💜 Building relationships outside of the clinic is so important to the Muenzer MPS Center as these relationships shape not just better care, but a stronger community for everyone impacted by MPS.🫱🏼‍🫲🏽🩺🧬