UNC Muenzer MPS Center

04/02/2026

Update on UX111 for Sanfilippo syndrome Type A (MPS IIIA):

The U.S. Food and Drug Administration has accepted the resubmitted Biologics License Application (BLA) for UX111, an investigational AAV gene therapy, and has set a PDUFA action date of September 19, 2026.

🔗 Read the full announcement: https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-us-fda-acceptance-bla-resubmission-ux111

04/01/2026

💙🧬 In the News: Advancing Awareness for Hunter Syndrome 🧬💙

We’re proud to share national coverage from NBC News highlighting the recent U.S. Food and Drug Administration approval of a new treatment for children with Hunter syndrome (MPS II)—an important moment for the rare disease community.

The article features insight from Kim Stephens, DBA, Executive Director of the Muenzer MPS Research & Treatment Center, along with perspective from Joseph Muenzer, MD, PhD, Director of the Muenzer MPS Center and Bryson Distinguished Professor, whose decades of work have helped move the field forward.

🗣️ “This is an important step forward for patients and families affected by Hunter syndrome, particularly as we continue working toward addressing the neurologic aspects of the disease,” said Dr. Muenzer.

This milestone represents hope—but also a reminder that there is still more work ahead for individuals and families living with MPS.
🔗 Read the full article: https://www.nbcnews.com/health/health-news/rare-disease-hunter-syndrome-fda-approval-children-rcna265813

After a string of denials for rare disease treatment candidates, the FDA last week greenlit a drug for Hunter syndrome, a progressive illness affecting about 500 Americans.

03/31/2026

⚾💙 Last Call: MPS Awareness Shirts for MPS Families 💙⚾

The Muenzer MPS Research and Treatment Center is making a final call for MPS families planning to attend MPS Awareness Day at the Carolina Baseball game on Tuesday, April 21, 2026, when the UNC Tar Heels take on High Point University.

🗓 MPS families must register by Wednesday, April 1 at 11:59 PM ET to guarantee your MPS Awareness shirt.

📅 April 21, 2026
⏰ First pitch at 6:00 PM ET
📍 Bryson Field at Boshamer Stadium
235 Ridge Road, Chapel Hill, NC

🎟️ MPS families will receive complimentary tickets and MPS Awareness shirts

👉 Register here: https://tinyurl.com/UNCMPSBaseballGame
We look forward to coming together as a community and showing strong support for MPS awareness.

03/30/2026

A significant milestone for the MPS community.

The FDA has approved AVLAYAH™, a new treatment for individuals living with Hunter syndrome (MPS II).

“This approval represents a breakthrough advance as the first therapeutic innovation for the Hunter syndrome community in nearly 20 years,” said Dr. Joseph Muenzer.

As the first FDA-approved therapy designed to cross the blood-brain barrier, this advancement has the potential to change how patients with MPS II are treated and offers new hope for families.

We are grateful to the patients, families, researchers, and advocates who made this progress possible.

Read the full press release:
https://investors.denalitherapeutics.com/news-releases/news-release-details/denali-therapeutics-announces-us-fda-approval-avlayahtm

03/26/2026

⚾💙 Friendly Reminder: Join Us for MPS Awareness Day at the Ballpark 💙⚾
The Muenzer MPS Research & Treatment Center is excited to bring our community together for MPS Awareness Night at a Carolina Baseball game as the Tar Heels take on High Point University.

📅 April 21, 2026
⏰ 6:00 PM ET
📍 Bryson Field at Boshamer Stadium

🎟️ MPS families will receive complimentary tickets and free shirts so we can show our presence together in the stands.

👉 Register: https://tinyurl.com/UNCMPSBaseballGame
We hope to see you there for a special evening of community and awareness.

03/25/2026

The Muenzer MPS Research & Treatment Center team recently came together in Chapel Hill for a team retreat.

It was a great opportunity to connect in person, share ideas, and strengthen the collaboration that drives our work forward each day.

We’re grateful for this dedicated team and the shared commitment to improving care and advancing research for individuals and families impacted by MPS.

03/19/2026

What is Glycogen Storage Disease (GSD)?

GSD is a group of rare genetic disorders that affect how the body stores and uses glycogen, often leading to low blood sugar and serious organ complications.

At the MPS Journal Club meeting at the Muenzer MPS Center, Dr. Monika Williams shared an overview of GSD Type I, including early signs like hypoglycemia and hepatomegaly, long-term risks such as kidney disease and hepatic adenomas, and how treatment approaches continue to advance.

From cornstarch therapy to emerging gene therapy, this work highlights how far the field has come—and how important coordinated, multidisciplinary care is for patients and families.
For a detailed look at the presentation, please visit: https://www.med.unc.edu/pediatrics/muenzer-mps-center/2026/03/gsd-type-i-presentation-highlights-from-dr-monika-williams/

03/18/2026

🚨 A recent Newsweek article highlights concerns from families facing potential loss of access to rare disease treatments following FDA decisions.

🔗 https://www.newsweek.com/mothers-terrified-as-children-could-lose-treatments-after-fda-denials-11671964

For those living with conditions like MPS, access to therapies is critical. Stories like these emphasize the importance of continued research, careful review, and collaboration to move treatments forward.

The Muenzer MPS Research & Treatment Center remains committed to advancing care, research, and support for the MPS community.

One mother told Newsweek she was "terrified" of losing access to the drug her daughter relies on as a result of an FDA rejection.

03/17/2026

Navigating an MPS diagnosis can be complex, but having the right team makes a difference.

In this video, Lindsay Torrice, MSN, CPNP-PC, Director of MPS Clinical Research, provides an overview of the Clinical Consultation Program at the Muenzer MPS Research & Treatment Center.

This program is designed to support patients, families, and providers with expert guidance, coordinated care, and individualized recommendations to help guide next steps.

Watch here: https://www.youtube.com
MPS Contact form: https://unc.az1.qualtrics.com/jfe/form/SV_efkRtvbohN1EDAO
s

An overview of the MPS consultation services, including multidisciplinary reviews, care coordination, and education—plus how families can get started.

03/11/2026

Behind the work of the Muenzer MPS Research & Treatment Center is a powerful personal story.

In this video, Dr. Kim Stephens, Executive Director of the Center, shares her journey as a mother of a child living with Hunter syndrome (MPS II) and how that experience shaped her commitment to helping families affected by rare diseases.

She is joined by Dr. Joseph Muenzer, Director of the Muenzer MPS Center at UNC School of Medicine, whose decades of research and care have helped advance treatments for patients with MPS.

▶️ Watch the video to learn more about the people and purpose behind the Muenzer MPS Research & Treatment Center.
https://youtu.be/C2DRdRhDP2g?si=oADwcJ9n8w0oRSWI

UNC Health Dr. Joseph Muenzer and Carrboro mom Kim Stephens, whose son Cole Mausolf has a rare genetic disorder called Hunter syndrome, are working together...

03/09/2026

⏳ 43 Days Until MPS Awareness Night at the Ballpark! ⚾💙

Join us as Carolina Baseball takes on High Point University for a special evening raising awareness for mucopolysaccharidoses (MPS).

📅 April 21, 2026
⏰ First pitch at 6:00 PM ET
📍 Bryson Field at Boshamer Stadium
🎟️ MPS families receive complimentary game tickets.

Register here:
https://tinyurl.com/UNCMPSBaseballGame

03/09/2026

Upcoming Webinar on Improving Care for MPS

Dr. Joseph Muenzer, Director of the Muenzer MPS Research & Treatment Center at UNC Chapel Hill, will be a featured speaker in the webinar “The Role of Managed Care in Improving Patient Care in Mucopolysaccharidosis (MPS).”

📅 March 10, 2026
⏰ 12:00–1:00 PM ET
🎥 Virtual webinar

The session will focus on improving care and access to treatment for individuals living with MPS and will highlight current and emerging therapies.

Learn more and register:
https://amcplearn.org/content/role-managed-care-improving-patient-care-mucopolysaccharidosis-mps-0?fbclid=IwY2xjawQam5pleHRuA2FlbQIxMQBzcnRjBmFwcF9pZBAyMjIwMzkxNzg4MjAwODkyAAEet6glSSNvqYlcT0yWtPYP_ra907xu4PvRQEv8clQeDCkMq0FZamIdiLeR3Gg_aem_ukfV5omxJirRHEujulCbjA -tabs-node-course-default1