10 Years of Hope, Research, and Relentless Advocacy

Together, we’ve built a global community, advanced research, and supported families every step of the way.
.

Together we’re proud to say:

We are ten years stronger. Ten years louder. Ten years closer to a cure.

Ten years ago, the words “H-ABC” were met with confusion, isolation, and heartbreaking uncertainty. For families like ours, there was no roadmap—no research, no community, and no path forward.

But we refused to stand still.

It’s hard to believe we are now in our tenth year of advocating for those living with H-ABC and TUBB4A-related leukodystrophies.

Together, we've made meaningful headway. The process is slow and complex—but every step brings us closer to treatments, to hope, and to a cure.

What Families Are Saying

‘To newly diagnosed families: this is your village. The Foundation to Fight H-ABC helped get us there.’

— Parent of a 6-year-old with H-ABC

Research Milestones

  • Sponsored research for AAV gene therapy (now in Year 4)

  • Pending rollout of drug repurposing study

  • Supported children receiving N=1 ASO treatment

  • Initiated TUBB4A Natural History Study

Family Support & Advocacy

  • Created national family services database

  • ULF Ambassador partnership

  • Global support for newly diagnosed families

  • Bilingual website launched

Awareness & Fundraising

  • Raised up to $100,000 annually

  • Hosted conferences & webinars

  • Promoted awareness through newsletters and media

  • Strong advocacy with industry and rare disease partners