Guest Voice: 10 years after my son’s desperate plea to the FDA
Our community's drive for something better changed today's first conversations
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On April 25, 2016, my son Billy stood at a podium in a hotel ballroom outside Washington, D.C., and told a room full of advisers from the U.S. Food and Drug Administration (FDA) something no 15-year-old should have to say out loud.
“FDA, please don’t let me die early,” he said.
This September marks 10 years since the pleas of Billy, his peers, their parents, and physicians helped lead to something extraordinary: The FDA approved Exondys 51 (eteplirsen) — a therapy Billy had already been receiving for five years in a clinical trial — as the first-ever treatment for Duchenne muscular dystrophy.
I think about this anniversary often — about how hard the road to approval was, but mostly about everything that’s happened since. A decade feels like a lifetime in a disease that, not so long ago, rarely let boys see much of their teenage years at all.
What 10 years actually looks like
Billy joined the eteplirsen trial in 2011 as one of only 12 boys nationwide given a chance on a drug that didn’t even have a name yet. It was just a glimmer of hope when we had nowhere else to turn.
I remember, a couple of years in, watching him open a Gatorade bottle by himself and run down a dirt path without falling. By the time Billy testified in 2016, the trial had ended, but he was still visiting the hospital every week — walking in on his own — for his infusions. These are small things, but also enormous things.
The FDA approved eteplirsen on Sept. 19, 2016. I found out before Billy — he was in school that morning — and I remember exactly how I felt. Something close to relief, and something close to victory, for him and for every other family who’d been waiting anxiously for the FDA’s decision.
The years since gave us moments I didn’t let myself plan for when he was younger — Billy walking across the stage at his high school graduation, starting college while still walking, and still going. Every one of those milestones sits on top of a decade of medicine doing exactly what we’d hoped it would.
Why I’m telling this story again
Approaching the 10-year anniversary of FDA approval feels like the right moment to reflect and dream about the decade ahead. Because Billy’s story — and our collective fight against Duchenne — is far from finished.
Duchenne is still a stubborn, complicated disease that doesn’t behave like more common conditions or fit neatly into the typical clinical trial mold. Ten years in, we’re still learning how to measure what it takes from a boy and what a therapy gives back.
Right now, the FDA is reviewing whether to convert two of eteplirsen’s sibling therapies, treatments for boys with different Duchenne mutations, from accelerated to traditional approval. Other Duchenne therapies are facing critical decision points, too.
If there’s one thing I hope the agency has learned over the past decade, it’s that a disease like Duchenne can’t be judged by a single test on a single day. We’ve had to track Billy’s progress over years and across everything we’ve watched him do. A couple of numbers on one chart never told his full story.
Some families today feel the way we did in 2011. They are clinging to hope. Others may not realize the therapies that keep their sons walking could be at risk if these reviews don’t go in their favor.
Billy went from an eager clinical trial participant to proof of what this class of medicine can do when boys get the chance to be on it. That’s why our family remains so driven to advocate, not because I think one family’s story should decide FDA policy, but because every family facing a Duchenne diagnosis deserves the chance he received.
Somewhere, another parent is having their first conversation about Duchenne right now. It’s still full of fear and questions, but it’s different than the one we had in 2011 because of this community’s drive for something better. We hoped. We testified. We enrolled. We pushed. We raised our voices and shared our stories. Together, we changed what it means to hear that diagnosis for the first time.
The next boy standing at a podium — and everyone who never has to — will stand on the progress of this last decade. Let’s ensure the next one holds even more promise.
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Note: Muscular Dystrophy 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 Muscular Dystrophy News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.
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