In the FA community, too many of our stories remain incomplete
There's both heartbreak and hope in what remains unwritten
I intended to publish a column in August that brimmed with hope and included a passage by my friend and fellow columnist Elizabeth Hamilton. We wrote about vatiquinone, which was up for approval with the U.S. Food and Drug Administration (FDA). It had the potential to become the second FDA-approved treatment for people ages 16 and older with Friedreich’s ataxia (FA), and perhaps more significantly, the first approved therapy for children with the condition. Elizabeth, who parents a child with FA, and I were optimistic, imagining a future that included two FA treatment options.
But that moment didn’t come.
On Aug. 19, the FDA declined to approve vatiquinone, citing insufficient data supporting its effectiveness.
And so, my August column never ran. For now, that story remains incomplete.
Pages left blank
Incomplete — like so many stories in our community. They begin filled with promise but too often end in heartbreak.
One story that ended all too soon belongs to my friend and fellow FA advocate, RJ Mercure. He passed away recently, just a year younger than I am. His contagious laugh, his poignant humor, and especially his vivid description of FA as “a giraffe in high heels on a windy day” remain vivid in my mind. Yet I don’t have closure, because his life ended before he could fully write it.
His giraffe metaphor is delightfully silly, but achingly accurate. It captures how we with FA exhibit grace under pressure: standing tall, yet unsteady; bold, yet vulnerable. It’s a truth we live every day.
RJ’s passing is a painful reminder: With FA or any rare disease, tomorrow isn’t guaranteed. Too many stories remain unfinished, too many pages remain blank. The condition imposes a humility that’s devastating but undeniably real.
What’s next?
Still, I refuse to let the story end here.
Yes, the FDA’s decision means vatiquinone will need to go through additional trials before its developer, PTC Therapeutics, can resubmit the application for its approval. But the door isn’t shut. PTC is working with the FDA to determine the next steps in the drug’s development.
And yes, RJ’s story ended much too soon, but his legacy continues. His analogy for FA reminds us to keep standing tall, to keep hoping.
We grieve RJ because his life mattered and his story is beautiful. We advocate for the approval of vatiquinone because the children in our community deserve their own stories — not blank pages, but chapters full of possibility.
Our stories, marked by setbacks and losses, aren’t over. We will write more. We will hope more. We will finish more.
Because living with FA means living with uncertainty. But it also means living with resolve. With community. With purpose.
There’s heartbreak in the unwritten. And yet there’s profound beauty in what remains possible.
Note: Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Friedreich’s Ataxia News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Friedreich’s ataxia.
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