With more work, research, and clinical trials, we know [a therapy] is coming

Opening ADVANCE 2023, Glenn O’Neill, President & Co-Founder of Cure Sanfilippo Foundation, shared the following remarks with the audience of more than 370 parents, researchers, clinicians, therapists, educators, and more, reviewing the 10-years of the Foundation and how the community working together has powered the impact it has made.

Glenn’s remarks

“Hi, I’m Glenn, president of Cure Sanfilippo Foundation.

Thank you for joining us for ADVANCE 2023 Sanfilippo community conference. We have another great group joining us. We have parents and families of children affected with Sanfilippo, doctors, scientists and researchers, therapists, industry leaders, and many others … thank you all.

Thank you to the speakers, who are taking their time out to help inform all of us. Thank you to our internal team at Cure Sanfilippo for the hard work on this conference. Thank you to our generous sponsors who help make this conference possible and free of charge for all attendees. Event sponsors are: JCR Pharmaceuticals, Denali Therapeutics, Novel Pharma, GC Pharma, Orchard Therapeutics, and Ultragenyx. We are glad to partner with you and thank you for your work in the Sanfilippo research space.

Before we get into the conference, I wanted to say a few words about our journey as a family and as a Foundation. Just over 10 years ago, on July 17, 2013, our daughter Eliza was diagnosed with Sanfilippo syndrome. Ten years. It’s hard to believe. Like many of the families here today, we were in shock, stunned, and with just a few words from the geneticist, our life was turned upside down forever. We were in complete despair and heartbreak, and not sure where to turn. We reached out to parents who had children with Sanfilippo and had come before us, and they provided us valuable guidance and advice.

We started looking at the science and research. And thanks to my wife Cara, a pediatrician, she was able to quickly understand the landscape. It was immediately obvious that there was a need to accelerate and increase the amount of Sanfilippo research happening. More clinical trials were needed. And all of it would require much more funding.
We also realized that connecting to other families was critically important. To be able to share experiences, forge friendships, and to just feel somewhat normal.

In an effort to make a significant impact, we decided to jump full-in and in November of the same year, 2013, created Cure Sanfilippo Foundation.

We began to tell everyone we knew about our Foundation and about Sanfilippo. And began grassroots fundraising. Shortly thereafter, a video about our family’s story went viral and gained much attention, spread awareness, and raised over $2 million in funding for research and clinical trials.

Cure Sanfilippo Foundation was off like a rocket.

From there, we began to receive calls and emails from other families, many newly diagnosed at the time and many who are in attendance today, who wanted to share their stories and join the mission.

I remember it well. It was one family that called me on a Tuesday. Then two more families on Thursday of the same week. That is when the lightbulb went off: Families want to join this effort. We all want to fight back against this disease, and we want to connect with others in the community.

What started as one family, now 10 years later, Cure Sanfilippo Foundation is 120 partner families strong! And it’s thanks to every single one of these families and their supporters that over $20 million has been raised for research and clinical trials. So much work by so many of you, our partner families and supporters, to make this happen.

There were more video campaigns along the way, like Saving Carter, Save Connor, Help Simon and Saving Liv. Together have helped raise millions. There are families doing annual campaigns, every year, like Spencer supporters, Will Power, Cure for Carson, Do it for Declan, the Wacker family/Believe in G, and so many more. Though too many to name here, we are grateful for the difference you all are making.

There are kind individuals and Foundations that generously support the mission in a significant way each and every year.

And a shout out over these past few years to all the families sharing their stories on social media, like TikTok and Instagram, massively increasing awareness of Sanfilippo. It’s made such a difference in children being diagnosed.

This is how the critical research done by so many of the people here today gets funded: Everyone chipping in where they can and every single dollar adds up. It is really incredible how this grassroots work has funded now 50 research grants and projects around the world and has helped lead to 3 clinical trials for children. And more trials to come, if we can continue to raise the needed funding.

We are so impressed by so many newly-diagnosed and veteran families who are jumping in the fight, with renewed energy! Nearly every donation we’ve received in our 10 years has been “in honor” of a particular child with Sanfilippo. So it is thanks to the drive of these families and their supporters that Cure Sanfilippo Foundation has been able to accelerate research and trials over this past decade.

Besides awareness and funding, the connections these families have made are so important and valuable. This year, so far, there have been two in-person Cure Sanfilippo family gatherings in Florida and Minnesota. It’s just impossible to describe the love and emotion at these events. These friendships stay with you; they help you get through.

So there has been incredible progress over the years thanks to the generous support of so many. But, we still don’t have approved and available treatments for Sanfilippo syndrome.

With more work, research, and clinical trials, we know that day is coming.”

On-Demand Recordings Available

Recordings of ADVANCE 2023 sessions are available for on-demand watching. ADVANCE featured high-level collaboration, information sharing, and Sanfilippo-specific learning sessions. Topics included cell therapy, biomarkers for Sanfilippo, data sharing to advance therapeutic development, new treatment approaches, and much more. Access the recordings.