The children we represent face a rare, degenerative disease with no approved treatment — one that can steal mobility, speech, and independence before they reach middle school. But science is moving. And so are we.

Thanks to this community, we’ve already launched multiple research initiatives — from gene therapy development to disease modeling — and begun laying the groundwork for real therapeutic options. Now, we’re focused on accelerating progress and closing critical gaps — including preclinical studies and clinical trial readiness.

We started this foundation as two families fighting for our daughters. Today, we’re a growing movement — guiding research, connecting institutions, and redefining what’s possible.