CureJax

Sanfilippo is stealing our son’s childhood.

CureJax is raising awareness, funding, and advocacy for families fighting a rare, rapidly progressive neurodegenerative disease.

How you can help Read our story

Our Story

Jax is four years old.

He’s loud, vibrant, and full of life. He loves Paw Patrol, fire trucks, riding quads with us, being outside, and most of all — baseball. If there’s a ball to throw or dirt to run through, he’s in it. He’s happy. He’s loving. He’s ours.

Jax was diagnosed with Sanfilippo Syndrome Type B (MPS IIIB), a rare and progressive neurodegenerative disease. It steals skills. It steals time. And time is not something we have.

When we received his diagnosis, we had a choice: sit back and watch… or fight.

We chose to fight.

We fight for access.
We fight for treatment.
We fight for research.
We fight to remove the obstacles standing between children like Jax and the cures they deserve.

This site exists to help people understand what Sanfilippo is, why early action matters, and how you can stand with Jax — and families like ours — through awareness, advocacy, and fundraising.

We are not waiting.
We are building the path forward.

Jax

What is MPS IIIB (Sanfilippo Syndrome Type B)?

MPS IIIB, also called Sanfilippo Syndrome Type B, is a rare, inherited genetic disorder that primarily affects the brain.

Children with MPS IIIB are born missing an important enzyme that the body needs to break down certain natural sugars. Without that enzyme, these sugars build up inside cells — especially in the brain — and cause progressive damage over time.

How does it affect a child?

Children with MPS IIIB often appear healthy at birth and may develop normally at first. But in early childhood, parents may begin to notice developmental delays, speech delays, or behavioral changes.

As the disease progresses, children gradually lose skills they once had. They may develop severe cognitive impairment, sleep disturbances, hyperactivity, mobility challenges, and eventually require full-time care.

MPS IIIB is a progressive neurodegenerative condition, meaning symptoms worsen over time. It is life-limiting, and currently there is no cure. Treatment focuses on managing symptoms and supporting quality of life.

How you can help

Three actions make a real difference: donate, advocate, and share.

Advocate

Right now, children with rare diseases face regulatory hurdles that delay access to treatments and potential cures. The FDA has significant authority granted by Congress — and families deserve consistent, timely application of that authority.

We are asking supporters to contact their Members of Congress and encourage them to:

  • Hold the FDA accountable for consistent regulatory decisions
  • Ensure rare disease therapies are reviewed efficiently
  • Support policies that expedite safe treatment approvals
Find your Members of Congress

Share

Sharing our story helps others understand the urgency and builds community. You can also reach us directly through our Facebook page.

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