When two of our four children were diagnosed with Friedreich’s Ataxia, we quickly realized this wasn’t going to be one of those parenting journeys with a step-by-step guide to follow. There was no quick course to download. Instead, we were handed a rare diagnosis and a lot of unknowns.
Naturally, we started asking: What can we do? What’s out there? How can we help our boys—and maybe even help the next family who gets this news?
That’s what led us to clinical research. And while it felt like the right step, it’s not one we took lightly.
Observational trials are one thing. They collect data, monitor progression, and are easier to say yes to. But interventional trials? That’s different. You’re saying yes to a treatment that’s still being studied. You hope it will help, but the truth is, no one knows yet. That’s the point of the trial.
And when you’re making that decision for your child, the weight of it is real.
Saying yes to a trial was our way of saying, “We’re not just going to sit here and wait.” It was our way of getting into the ring as a whole family, even if we weren’t sure what the outcome would be. Think Rocky Balboa, but with more paperwork and blood draws and fewer black eyes. Because even if it’s hard, even if it’s uncertain, every piece of data moves the field forward. Every participant helps build knowledge that could lead to better treatments—not just for our kids, but for everyone living with FA. That matters.
Now, let’s talk logistics. Trial participation sounds noble—and it is—but let’s be honest: it’s not exactly fun or easy. There were early mornings (coffee: essential), long drives (thank you, road trip playlists), missed school days (sorry, teachers), and quite a few nights away from home. We’ve had to take some time off, tag-team responsibilities, and lean hard on friends and family (they are our heroes!). Site visits can last for hours, so it’s often a full-day affair—and one that requires snacks, chargers, and a solid sense of humor.
Still, every appointment reminded us: progress in rare disease research only happens when families are willing to show up, even when it’s wildly inconvenient and requires remembering where you parked in that giant parking garage… again.
Do we know what the future holds? No. But we know this: research matters. Participation gives us purpose. It reminds us that we’re not just along for the ride—we’re helping steer! We’re a small part of a much bigger story. And that brings REAL hope.
So, if you’re a fellow parent staring down this road, we’d encourage you to jump in. Yes, ask the questions, talk to the doctors, read the consent forms, and if it makes sense for your family—jump in. Because your story matters. Your data matters. And your willingness to say “yes” could help unlock what’s next.
Also, bring snacks.
Written by
Marc and Maggie Likins