2025 Hunter Syndrome Community Conference
March 8, 2025
Hunter Syndrome Community Conference
Hunter Syndrome Community Conference 2025
Join us for the 5th annual Hunter Syndrome Community Conference, a gathering dedicated to education, support, and connection for individuals and families affected by Hunter syndrome. This inspiring event features informative presentations from leading experts, healthcare professionals, and advocates in the Hunter syndrome community. Gain valuable insights into the latest research, treatments, and resources while connecting with others who share similar experiences. Whether you are a patient, caregiver, researcher, or supporter, this conference offers a meaningful opportunity to learn, share, and build a stronger community.
Don't miss this chance to come together, exchange knowledge, and find support in the journey with Hunter syndrome!
Speakers
Kristin McKay
President and Executive Director, Project Alive
Miriam Pedroza
MPS II Mother
Jenn Hanebury-Estevez
MPS II Mother
Julie Ferullo
MPS II Mother
Jeanette Henriquez
MPS II Mother, Vice Chair of Project Alive BOD
Barbara Burton, MD
Attending Physician, Genetics, Genomics and Metabolism, Lurie's Children's Hospital, Professor of Pediatrics (Genetics, Genomics and Metabolism), Northwestern University Feinberg School of Medicine
Kim Stephens, PhD
Executive Director, Muenzer MPS Research & Treatment Center
Leigh Anne H. Weisenfeld, MSW, LCSW
Clinical Instructor, Muenzer MPS Research & Treatment Center
Raymond Wang, MD
Director of the Multidisciplinary Lysosomal Storage Disorder Program at CHOC. Board certified clinical geneticist and biochemical genetics specialist.
Katey Hoffman, MD
Pediatric Hospitalist, MPS II Mother
Kyle Underwood, MHA
Chairperson of the Board, Project Alive
Joseph Muenzer, MD, PhD
Bryson Distinguished Professor of Pediatrics, Pediatric Geneticist and Researcher, Director of Muenzer MPS Research & Treatment Center
Schedule
March 08
MPS II 101
Join Katey Hoffman, MD, a parent to a son with Hunter syndrome and a Pediatrician, to take a deeper dive into Hunter syndrome. Katey will explore symptoms, specialists, treatments, and more to provide an understanding of this complex diagnosis. This is a great session to encourage your teachers, providers, babysitters, and anyone else in your life to join!
Neurocognitive Testing
Leigh Anne Weisenfeld joins us from the Muenzer MPS Research & Treatment Center to discuss neurocognitive testing. Children with Hunter syndrome undergo these tests frequently whether in a developmental clinic, a clinical trial, or at school. Leigh Anne will explore the different types of tests, best practices for conducting these tests for children with MPS II, and helpful tips for caregivers. This session will be beneficial to both caregivers and providers administering neurocognitive or developmental tests.
Blood Brain Barrier Challenges & How Science is Advancing Treatment
Dr. Burton is a leading expert in MPS and has participated in countless research for Hunter syndrome. In this session we will gain a better understanding of the blood brain barrier, why it is so challenging to get medication to the brain, and how researchers are tackling this issue. Dr. Burton will also discuss novel enzyme replacement therapies that aim to cross the blood brain barrier.
MPS II Biomarkers
Dr. Joseph Muenzer, a world renowned expert in MPS, will discuss biomarkers in MPS II. With the FDA's Accelerated Approval Pathways, biomarkers (or surrogate endpoints) have become a frequent topic of conversation. In this session we will learn what biomarkers are, how they are being used in the clinical trial process, and which biomarkers are assessed in MPS II.
Gene Therapy in Hunter Syndrome
Gene therapy is a complex scientific breakthrough now being studied in Hunter syndrome. With two applications in a completed or enrolling stage of the clinical trial process, now is the time for us to take a deeper dive into this science. Dr. Raymond Wang, a Pediatric Metabolic Geneticist, will provide a detailed explanation of gene therapy and the specific applications in Hunter syndrome.
Clinical Trials 101
Clinical Trials 101 will provide a clear and comprehensive overview of the clinical trial process, breaking down key terminology, the different trial phases, and how study endpoints are determined. Attendees will also learn about the FDA's role in drug development and the new Accelerated Approval Pathway. Whether you're new to clinical trials or looking to deepen your understanding, this session will equip you with the knowledge to navigate the process with confidence.
Caregiver Panel: Treatment Tips & Tricks
This Caregiver Panel brings together four moms of children with Hunter syndrome to share their experiences, insights, and what they wish they had known before starting treatment. With perspectives from both clinical trials and stem cell transplantation, this discussion will provide practical advice, lessons learned, and tips for navigating the treatment journey. Moderated by Kristin McKay, this session offers a candid and supportive space for caregivers seeking real-world guidance.
MPS II Advocacy Landscape
This session will explore the ongoing and upcoming advocacy efforts shaping the future of Hunter syndrome care, research, and policy. Presented by Kim Stephens, PhD-an MPS II mom, former President of Project Alive, and a leader in advocacy-this session will highlight key initiatives, legislative priorities, and ways the community can take action. Attendees will leave with a deeper understanding of the advocacy landscape and how they can contribute to meaningful change.
Unmet Needs in MPS II Survey Results
Kristin McKay will present key findings from the recent survey conducted by Project Alive in partnership with Denali Therapeutics. This session will explore the biggest challenges facing individuals with Hunter syndrome and their families, highlighting gaps in care, treatment, and support. The session will provide insight into how these results can drive future advocacy, research, and policy efforts to better address the needs of the MPS II community.
Our Sponsors
Hope Champion
