Our son Jax is 5 years old. In February, he was diagnosed with Sanfilippo Syndrome Type B.

Sanfilippo is a rare, progressive disease that slowly damages the brain and body. It is sometimes called “childhood dementia” because children often develop normally for a while and then begin losing the skills they worked so hard to learn.

The short version is this: Jax’s body cannot properly break down certain waste products, and that buildup causes damage, most of it in the brain.

Jax is still the same happy little boy everyone knows and loves.

He smiles, laughs, plays, and brings joy everywhere he goes. He has no idea what is happening inside his body. And from the outside, it is not always obvious that anything is wrong. That is one of the cruelest parts of Sanfilippo. The damage starts long before anyone can see it.

That is why we cannot wait.

Multiple Sanfilippo Type B clinical trials are coming soon, and they are fully funded. These are not distant ideas, and they are not vague possibilities. They are real treatment programs moving forward — designed to go after the disease at its source, with the hope of slowing it down, stopping it, or possibly changing the course of the disease altogether.

Sanfilippo primarily damages the brain. Once brain function is lost, it may not come back. That means the condition Jax is in when a trial opens could matter enormously. Every month of brain function we can protect now may give him a better chance later.

A treatment can only work with what is still there.

That is what this fundraiser is for.

We are not asking for help paying for a clinical trial. The trials are funded. We are asking for help keeping Jax as healthy, strong, and stable as possible so that when a trial door opens, he is in the best condition we can get him in to walk through it.

Since Jax’s diagnosis, we have spent every spare hour learning, asking questions, talking to doctors and researchers, and trying to figure out what may help. There is no playbook for this. We are making the best decisions we can with what we know.

None of this is simple, and none of these supportive therapies are being treated like magic answers. We know they are not cures. But we also know Jax’s brain and body need help right now. While we wait for the clinical trials that are coming, we are trying to give him every possible advantage. With Sanfilippo, lost time matters. Lost brain function matters. So we are doing what parents do: trying to protect what we still can.

Funds raised will help with things like:

This is one of the most humbling places a parent can find themselves. We are learning that love, effort, and determination are not always enough by themselves. The limits of what we can do on our own do not match the size of what Jax needs.

We will keep doing everything within our power. But many of the therapies, consultations, travel costs, equipment, and supports that may help him are not covered by insurance. That is why we are asking for help — not because it is easy, but because giving Jax the best chance matters more than our pride.

Jax is still here. He is still happy. He is still fighting, even though he does not know there is a fight.

And we are going to fight with everything we have.

If you are able to give, thank you. If you are not able to give, sharing this fundraiser means more than you know. Prayers, encouragement, connections, and help spreading Jax’s story all matter.

Thank you for loving Jax and standing with our family.

With love,

The McConnell Family