Finding hope in a WORLD of research

Last week, Trevor, Simon and I had the privilege of attending the WORLD Symposium in San Diego — and I’m still processing all that it meant. So grateful to have shared the experience with our friends and fellow Morquio-parents Helen and Bobby Vice – the incredible forces behind Rooting for Robert in Louisville, Kentucky.

As Simon’s mom and the founder of CureMPS, so much of my work happens in my head, in emails, medical appointments, hospital hallways, at the family dinner table and during fundraising events like the Hoedown for Hope. But at the recent WORLD Symposium, I stepped into a different kind of room — one filled with researchers, clinicians, biotech leaders, and families from around the globe, all united by rare disease.

It was both humbling and electrifying.

One of the best parts for us was spending time with the research team from UCSF Benioff Children’s Hospital – they’re behind the clinical trial of vosoritide that our CureMPS research fund (through our charitable partner The Isaac Foundation) is helping fund. To build friendships with the researchers and clinicians who are dedicating their careers to kids like Simon — and to know that our community’s fundraising is helping move that work forward — was deeply impactful.

These aren’t abstract grants or distant research lines in a report. These are real people, in real labs, working urgently and intentionally toward better outcomes for our kids.

We also had the chance to meet Dr. Joey Skeate from the University of Minnesota’s Centre for Genome Engineering. They are the team of genetic engineers pursuing an innovative T-cell micropharmacy project. It’s a promising breakthrough treatment for MPS and other lysosomal storage disorders and with funding from our CureMPS Research Fund, we’re helping open the door to using it for MPS IVA. Hearing firsthand about this novel approach — reprogramming cells to deliver therapy in a targeted, precise way — felt like stepping into the future of medicine.

It’s ambitious. It’s cutting-edge. And it’s exactly the kind of thinking rare diseases deserve.

What struck me most throughout the conference was the alignment between families and scientists. There is no separation between urgency and innovation — they fuel each other. Families bring the “why.” Researchers bring the “how.” Together, we build the “what’s possible.”

Meeting up with our research partners and meeting new ones reminded me that progress in rare disease doesn’t happen in silos. It happens when philanthropy, science, advocacy, and lived experience intersect.

Walking through the conference halls in San Diego, I felt something shift. The work we are doing at CureMPS — the Hoedown, the community partnerships, the late nights writing blog posts — it’s not small. It’s connected to a global movement. It’s accelerating real science.

And perhaps most importantly, it’s building relationships with the very people who are working to change the trajectory of Simon’s life.

We came home tired — but filled with renewed conviction.

Hope is not passive. It’s built in labs. It’s funded in fairgrounds. It’s strengthened in conference halls. It’s carried by community.

And we are right in the middle of it.