Meet the Cure ALD Family

Our ALD story began in 2024 when Terry, pregnant with our second child, took a carrier screening test and tested positive for ALD. She’d screened negative in 2018. We learned shortly after that Theodore, three at the time, also has ALD. The emotions came fast—anger at systems that nearly missed this, powerlessness reading about treatment options that are invasive and risky, the particular sadness of knowing your beautiful child carries something he doesn’t deserve. But here’s the paradox that changed everything: we’re incredibly grateful we caught it early. Most families in the ALD community aren’t that lucky. Early detection gave us something families diagnosed after symptoms appear will never have—time to explore options, time to plan, and time to accelerate research toward a cure before damage occurs. That distinction is everything. It’s the difference between hope and crisis intervention. We know enough about ALD to know it can be cured. Gene therapy is working—Skysona, the first FDA-approved gene therapy for ALD, is showing efficacy in early-diagnosed children but with risks. Researchers have mapped biomarkers that predict disease progression. Clinical trial networks exist. The roadmap is drawn. Ten years ago, this would have been fantasy. Today, it’s fact. What’s missing isn’t brilliance. It’s speed—the coordinated funding that transforms a decade-long cure timeline into five years or less, the resources to run multiple clinical trials in parallel, the research investment that tells scientists “Yes, ALD matters. Move as fast as you can.” Right now, ALD research is scattered, funding is fragmented, and federal dollars are under pressure. The reality is simple: private funding now determines the cure timeline. We created Cure ALD to address two gaps. First: insufficient research funding for cure-focused innovation. We have the science and the scientists, but not the coordinated private capital moving at the speed this moment demands. Second: newly diagnosed families deserve immediate access to a community, to mentor families further along, to clear answers about what’s possible. The gap between diagnosis and confident decision-making can span months of research, confusion, and fear. Here’s what we’re asking: help us decide when. We’re not asking for traditional donations. We’re asking you to become part of the fastest-moving ALD cure timeline in history. Create your own fundraising page and challenge your network—your college friends, your colleagues, your running group. Your voice reaches people ours doesn’t. Every $5,000 helps fund a graduate student researcher for a year. Every $25,000 accelerates a clinical trial timeline by months. Every $100,000 unlocks a gene therapy safety study. When you create a fundraising page, you’re signaling to researchers that ALD matters, that the community is mobilized, that speed is possible. Together, we’re compressing a decade into years—the difference between Theodore getting his cure as a child or as an adult, between newly diagnosed children today avoiding the worst outcomes entirely. The roadmap exists. The community exists. Now we have you.