04/10/2026
READ THIS UPDATE!!!!!! THIS IS THE BEST NEWS OF THE YEAR SO FAR!!!!!
Okay, I’ve started and restarted this caption so many times, because this update is huge and complicated and I want to explain it clearly.
Most of you know that back in November, the Sanfilippo Type B community rallied around an Expanded Access Program. That was the urgent need in front of us. We had one month, a huge goal ($3.8 million!), and a treatment path that could help buy our children time.
That EAP is for enzyme replacement therapy. It is not a cure. We’ve said that from the beginning. But it is still incredibly important, because once it’s available, it gives many children a chance to stop progression sooner rather than later. For families like ours, that is not small. That is everything. That program is still in development and we are incredibly thankful to this community for making that possible.
At the time, that was the bridge in front of us. And you all helped build it.
***
But since December, a whole lot has been happening behind the scenes.
In February, we found a groundbreaking gene therapy program out of France and started asking a new question: is there any real path to help bring this to the U.S.? And if so, how can we help it move forward faster?
That plan is what we now lovingly call Project Poppy. 💜
Project Poppy is a strategy to help expand this French gene therapy program into the U.S. as quickly as possible by pursuing an N=1 path, with Poppy as the first patient treated. In simple terms, an N=1 approach allows for a more expedient development pathway to reach the clinic faster for children who don’t have time to wait, while also helping establish the scientific, manufacturing, and regulatory groundwork that could open the door to others. But this pathway requires that a single child go first — to say yes so that other children may benefit much sooner than a typical regulatory path would allow.
And just for clarification: while this pathway begins with one child, the vision has never been about one child alone. The reason to pursue this approach is to create a remarkably faster, more scalable path for children with Sanfilippo Type B to follow. In other words, one child goes first so that others do not have to wait as long behind her.
And that matters because this is different from an ERT. The ERT is a bridge (a necessary and important one, and a massive win for these kids). But gene therapy is the closest thing we can currently see to a real long-term answer. It may not undo the damage already done, but its goal is much bigger than buying time. Its goal is to stop this disease at its source in a lasting way.
***
And now for the part that still does not feel real to type:
Project Poppy is happening.
AND
Project Poppy is fully funded.
The biggest answer to “how can we help it move forward faster?” Funding. This project — the existing French trial and the ability to bring this treatment to the US — would need about $4 million. But what no one else knew, was that behind the scenes, that exact amount of money was already ready at a foundation, ready to go. Because you see, families didn’t just meet the $3.8 million goal in November, we blew right by it. When we announced in December that we’d met our fundraising goal (and then some), there was actually money still coming in. $4 million more. The exact amount needed to move this project forward.
Which means the same Type B community that came together to fund the first-ever Expanded Access Program for Type B also *unknowingly* helped fund the first gene therapy pathway for Type B. It’s hard to even type it without crying.
You all gave back then knowing you were funding a bridge. A desperately needed bridge, but not a cure. Now we can see that same generosity *also helped fund the most meaningfully curative path we could actively pursue.* UNREAL. You helped fund time. Readiness. AND you helped make sure that when the next door opened, this community could walk through it. 🥹
I want to especially honor so many people who played significant roles in this story:
💜 Families who advocated and fundraised, who are loving these children every hour of every day.
💜 Donors who gave sacrificially and in incredible faith.
💜 Doctors and researchers working for years on these answers.
💜 The people working behind the scenes on Project Poppy, making calls, asking questions, solving problems, carrying pressure, and refusing to give up.
This has not been easy and we would not be here without any of you.
But even with all of that being true, we also have to say this: we could not have written this story in a million years. And I don’t think we could ever replicate it if we tried.
But God.
This whole process has been bathed in prayer from the very beginning, and we truly believe we are watching God answer those prayers right in front of us.
So yes, we are deeply thankful for every share, every donation, every meeting, every email, but we are especially thankful for every prayer, because mountains are moving, and that is not our doing.
We are trying to save lives — we don’t want to see another child lose their life to this disease.
We are trying to save *our own children’s* lives. We want Poppy and Oliver to have a future, and we are asking God for that boldly.
But most importantly of all, we want people to hear the name of Jesus through our story. Because as much as we love our children — and we would do anything to save them — Jesus loves more deeply, more perfectly, and more completely than we ever could.
As miraculous as this story feels to us, the greatest hope we have to offer anyone is still not gene therapy, funding, or medicine.
It is Jesus.
So today, we are celebrating.
We are grateful. We are overwhelmed. We are hopeful. We are in awe of what has happened and of the people God has used to get us here.
And if you take anything from our family’s story, I hope it is not just that Poppy has a path forward.
I hope it is that Jesus is real. That He is near, that He hears prayers, and that He is still moving mountains.
Project Poppy is funded.
All glory to God. 💜💜
