I'm 25 with a terminal disease. We need an FDA commissioner that hears people like me

People with Duchenne muscular dystrophy (DMD) aren't expected to see another birthday after this one. DMD is a progressive disease; my muscles weaken over time and things that used to be automatic now take planning, assistance, or simply aren't possible anymore. Even sleeping can be hard. Bed sores are now an issue. Basic physical function takes work most people never have to think about.

I know what the statistics say. I am not ready to let them write the ending of my story.

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I want to grow my career, I want to get married and start a family. I want to wake up decades from now and realize that turning 25 wasn't the beginning of the end — it was just another birthday I was blessed enough to reach.

That's why I'm watching the Senate confirmation fight over Dr. Heidi Overton, the nominee to lead the FDA, more closely than most 25-year-olds ever watch Washington.

For most Americans, an FDA commissioner is just another name in the news. For the 30 million Americans like me living with a rare disease, that person can decide how many options I have and how much time I have to use them.

To be clear: I'm not asking the FDA to abandon science. I respect doctors, clinical trials and hard evidence. I know some drugs don't work and some carry real risk.

But there's a question Washington doesn't ask often enough: who gets to decide how much risk a patient is willing to accept?

If I understand the risks, my doctor understands the risks, and there's real evidence a treatment could help, why does a regulator in Washington get the final word on whether I'm allowed to try?

Congress already recognized part of this principle when President Trump signed the Right to Try Act into law in 2018. That law was a major step forward because it affirmed something patients facing life-threatening diseases have always known: when approved options are exhausted, and a doctor believes an investigational treatment may help, patients should not be treated as passive observers in their own survival.

Right to Try was not the end of that idea. It was the beginning of a patient-centered philosophy that should shape the FDA’s culture more broadly. The same principle should apply when the agency weighs evidence, risk, urgency and access for rare-disease patients. If the patient understands the risk, the physician understands the risk, and there is real evidence that a treatment could help, the system should lean toward partnership instead of paternalism.

That’s why Senator Ron Johnson and Representative Diana Harshbarger introduced Right to Try 2.0: to carry that same principle into the next era of medicine, including individualized treatments that do not fit neatly into the FDA’s traditional approval model. They are right to press the issue. But the next leader at the FDA should not have to wait for Congress to force a more patient-centered approach.

The FDA already knows that time matters. Its accelerated approval pathway exists precisely because making patients with serious, life-threatening diseases wait years for a traditional clinical endpoint can cost them the thing they can't get back. The agency has used that pathway for multiple DMD treatments already.

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Because waiting is never neutral when you have Duchenne. Every year can mean another loss of strength, another limitation, another piece of independence gone for good.

My brother, who also lives with DMD, has seen the other side of this fight, too. Even when a treatment exists, actually getting it can be its own battle. Traveling hours away from home for a multiyear clinical trial sounds manageable in a Washington conference room. It looks very different when you're the one making that trip while your body is working against you as opposed to having access to the therapy at your own hospital.

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That's why I want the next FDA commissioner to listen to patients like me. We don’t just read about the disease in books or look at it through a microscope — we live with it every day. It’s in us — woven into our DNA. Living with Duchenne has forced me to learn more about drug development and regulatory pathways than I ever expected to know.

I want an FDA that works with patients, physicians and rare-disease experts. That's also why I'm going to say something people will have opinions about: I hope whoever leads the FDA brings on Dr. Houman Hemmati as deputy commissioner. He has spent his career in biotech, not politics, building treatments for rare and degenerative diseases instead of just regulating them from the outside. He's also my friend. I know what that sentence invites. Critics will say I'm just trying to get someone I know into the room. They're right that I know him. What they're missing is: he doesn't just know my disease, he's lived it with me.

I'm not asking anyone to promise me forever. I'm asking for the chance to fight for the future I still see.

I've already outlived what a lot of people expected. I'm not ready to stop.

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Senators considering Dr. Overton's nomination have a choice to make too: confirm a commissioner who will treat patients as partners in their own survival, or keep a system that treats us as bystanders to it.

It's time. And I'd like Washington to finally understand what mine is worth.

Elijah Stacy is a bestselling author, public speaker, Hollywood writer and producer, biotech consultant, and recognized Key Opinion Leader in the fight against Duchenne Muscular Dystrophy.