Building a Future Together

A MPS II COMMUNITY CONFERENCE

BUILDING A FUTURE TOGETHER

A MPS II COMMUNITY CONFERENCE

BUILDING A FUTURE TOGETHER

Join Project Alive for a day of learning, connection, and fun for the Hunter syndrome community! This event will be held on Saturday, May 3, 2025 at the Rizzo Center in Chapel Hill, NC followed by a tour of the MPS Center on Sunday, May 4, 2025.

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Event Details

About the Conference

Project Alive is thrilled to host the Building a Future Together conference on May 3rd, 2025. The conference will bring together families impacted by MPS II, clinicians and other providers, industry partners, and advocates. Families will hear from industry, learn about topics that frequently impact the community, and network with their peers. Families of other MPS types are welcome to join us using the same MPS family ticket options.

Following the conference, attendees will have an opportunity to join us on May 4th at the Muenzer MPS Research and Treatment Center for a tour of the facilities.

Room Block

There is a room block at the Rizzo Center for all guests to utilize at a discounted rate. The room block is available for the nights of May 2nd and 3rd. You must book by April 11th to utilize the discounted rate. There are King and Double Queen rooms along with ADA rooms available. If you'd like to extend your stay, we recommend you call the hotel with the room block information to have them assist you with your reservation.

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Scholarships

Scholarships are available for families impacted by any form of MPS. Scholarships will include the full cost of airfare (or mileage/rental car reimbursement), hotel stay for 2 nights, and conference registration for up to 4 members of the household. Applications will be reviewed by a panel to determine awardees. Priority will be given to families based on factors including having a child that has been diagnosed in the last two years, previous ability to attend any MPS conferences, and financial need. The number of families awarded will be dependent on available funding and costs per family for their travel. Scholarship applications will close on March 15, 2025. Awardees will be notified within one week. Awardees will be allotted one week to confirm their acceptance before their award is released to families on the waitlist.

Scholarship Application

Translations & Closed Captioning Services

Live translations and closed captioning will be provided through a mobile app. Please see our registration table at the conference and scan the QR code to access these services. At this time, Spanish is the only available language. Please let us know if you need translations for a different language and we will do our best to accommodate.

Kids Camp

Child care is available at no cost to families impacted by MPS. Affected children and unaffected siblings are welcome to attend. Infants to teens welcome. There is limited availability for child care so please ensure you register early and complete all necessary forms. Registration for child care will close on April 21, 2025.

Kids Camp will be hosted by our trusted partner, Beyond Blindness. Beyond Blindness is a nonprofit organization in Southern California with the mission to empower children with visual impairments and other disabilities to achieve their fullest potential. They provide early intervention (both in home and in a day class), preschool classrooms, robust therapy services, and more. Beyond Blindness has 60 years of experience caring for children of all abilities and have had children with MPS in their school! To learn more about their program, please visit www.beyondblindness.org.

SibShop

This year, our conference will feature a SibShop opportunity! This session will be geared towards unaffected siblings within the ages of 7-16. This session is free to all siblings that have a sibling with MPS, including bereaved siblings. SibShops are a fun way for siblings to connect, share their experiences, and learn helpful coping skills through a playful approach.

You can register your interest in SibShops when you place your conference order. You will receive an additional email for any specific information needed in order to complete the registration for SibShops.

MPS Center Tour

A tour of the Muenzer MPS Research and Treatment Center will be available on the morning of May 4th. You may select to add the tour when registering for the conference. Tours will last roughly 10-15 minutes and will be given in groups of 8-10 people. Your household will be able to do the tour together.

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Map

Kids Camp McLean Hall.png

Speakers

Jennifer Siedman

Director of Community Engagement, Courageous Parents Network

Nidal Boulos, Ph.D.

Senior Director, Clinical Science, REGENXBIO

William Bakker, Pharm.D.

Senior Medical Science Liaison, Denali Therapeutics

Robin LeWinder, PhD

USA Medical Affairs & Medical Communications Lead, JCR USA, Inc

Leigh Anne H. Weisenfeld, MSW, LCSW

Clinical Instructor, Muenzer MPS Research & Treatment Center

Kyle Underwood, MHA

Chairperson of the Board, Project Alive

Kristin Carleton

CEO, All Needs Planning

Kim Stephens, PhD

Executive Director, Muenzer MPS Research & Treatment Center

Jennifer L. Cohen, MD

Assistant Professor of Pediatrics in the Division of Medical Genetics at Duke University, PI for PEARL Clinical Trial

Nathan Grant, MPhil

President and Founder, Siblings with a Mission, Harvard Medical School Student

Kristin McKay

President and Executive Director, Project Alive

Schedule

May 03

7:00 AM - 7:45 AM EST

Registration Opens & Breakfast

7:45 AM - 8:00 AM EST

Welcome Address

8:00 AM - 9:00 AM EST

Financial Planning in a Rare Disease Family

Building Stability and Peace of Mind Through Every Stage of the Journey Financial planning is complex for any family—but for those navigating a rare disease diagnosis, it can feel overwhelming. From medical expenses and insurance challenges to long-term care, government benefits, and future planning, families face unique financial hurdles that require thoughtful and strategic guidance. In this session, Kristin Carleton of All Needs Planning will share expert insights tailored specifically for rare disease families. She’ll cover key considerations such as special needs trusts, ABLE accounts, guardianship and estate planning, and how to prepare for both expected and unexpected costs over time. Whether you're just beginning your journey or planning for adulthood and beyond, this session will provide practical tools and peace of mind as you plan for your family’s financial future.

9:00 AM - 9:45 AM EST

Behaviors in Hunter Syndrome

Understanding Challenges, Supporting Strengths, and Navigating Neurocognitive Changes Behavioral challenges are a significant and often misunderstood part of the Hunter syndrome journey. In this session, Leigh Anne Weisenfeld will explore the range of behavioral symptoms that can occur, how they evolve over time, and strategies for supporting individuals and families through these changes. From communication difficulties to sensory sensitivities, aggression, and anxiety, this session aims to foster greater understanding and offer practical tools for daily life. Leigh Anne will also share insights into neurocognitive testing—what it is, why it matters, and how it can help track changes, support school planning, and guide care decisions. Whether you're a parent, caregiver, or professional, this session offers valuable information to help you better understand and respond to the unique behavioral and cognitive needs of individuals with Hunter syndrome.

9:45 AM - 10:00 AM EST

Break

10:00 AM - 10:10 AM EST

DNL310 Study Presentation

10:10 AM - 10:20 AM EST

JCR Pharmaceuticals - Pabinafusp alfa: Treatment and Clinical Trial Update

Since its inception in 1975, JCR Pharmaceuticals has focused on the research, development, and manufacture of biotherapeutics. Our engagement in lysosomal storage disorders (LSD) led to the discovery and establishment of a blood-brain barrier penetrating technology, which has resulted in the first approved biotherapeutic in Japan designed to cross the blood-brain barrier to treat CNS signs and symptoms of MPS II. JCR Pharmaceuticals is now applying the same technology to multiple clinical programs in our LSD pipeline for Japan and the rest of the world.

10:20 AM - 10:30 AM EST

REGENXBIO’s Investigational Gene Therapy for the Treatment of Mucopolysaccharidoses Type II

10:30 AM - 10:40 AM EST

Takeda Pharmaceuticals

10:40 AM - 10:50 AM EST

Stem Cell Gene Therapies in MPS Diseases

10:50 AM - 11:00 AM EST

Interim Results from the PEARL Trial: Prenatal Enzyme Replacement Therapy

11:00 AM - 11:10 AM EST

Q&A

11:10 AM - 11:40 AM EST

Lunch

11:40 AM - 12:30 PM EST

So Many Decisions!

Parents of medically complex children have to make a myriad of decisions, big and small, every day. It's fatiguing. Understanding how you process decisions, and recognizing how your child's providers support your decisions can help mitigate feelings of overwhelm, fear of regrets, and create an environment where decisions can feel shared.

12:30 PM - 1:15 PM EST

Breakout: What I Wish I Knew Earlier

Real Talk, Shared Wisdom, and Support for the Journey Being a parent or caregiver in the rare disease world often means learning as you go—sometimes the hard way. This open, heartfelt session invites you to reflect on your journey and share the insights, tips, and “aha!” moments you wish you’d known earlier. Whether it’s navigating medical systems, advocating at school, managing daily care, or protecting your own well-being, your experience could be exactly what another family needs to hear. Come ready to listen, learn, and connect in a supportive, judgment-free space. Together, we’ll build a collective toolbox of practical advice and emotional support to lighten the load for others walking a similar path.

1:15 PM - 1:45 PM EST

The Journey in Aging with Hunter Syndrome

Exploring the Transition to Adult Care and the Evolving Needs Across the Lifespan As individuals with Hunter syndrome grow older, families face new challenges, questions, and milestones—many of which are not well understood or supported. In this session, Nathan Grant will present early findings from a recent survey exploring the transition from pediatric to adult healthcare in MPS, with a particular focus on Hunter syndrome. His research sheds light on what families and individuals are experiencing during this critical shift and identifies gaps and opportunities for better support. The session will also open a conversation about broader issues related to aging with Hunter syndrome, including long-term care planning, quality of life considerations, and the evolving needs of adolescents and adults. Join us to learn, share, and be part of shaping a more informed and compassionate approach to lifelong care.

1:45 PM - 2:00 PM EST

Muenzer MPS Research & Treatment Center Overview

Named in honor of Dr. Joseph Muenzer, a pioneer in the field of mucopolysaccharidosis (MPS), the Muenzer MPS Research and Treatment Center is dedicated to improving the lives of individuals with MPS through comprehensive care, cutting-edge research, and compassionate support for families. In this session, attendees will get an in-depth look at the Center’s multidisciplinary approach to diagnosis, clinical management, and long-term care for patients with MPS, including Hunter syndrome. You'll also learn about the Center’s leadership in clinical trials and research aimed at developing new therapies, as well as the educational and emotional support services offered to families navigating life with a rare disease. Whether you're new to the community or looking to understand more about current and future treatment options, this session offers valuable insights into the work of one of the world’s leading MPS centers.

2:00 PM - 2:10 PM EST

Closing Remarks

Schedule

May 04

8:00 AM - 10:00 AM EST

MPS Center Tour

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