A 25-year-old man living with Duchenne muscular dystrophy is pressing the Senate to confirm an FDA commissioner who will treat patients facing terminal illnesses as partners in their own survival rather than bystanders to it. In a first-person account published as the Senate weighs the nomination of Dr. Heidi Overton to lead the agency, he describes a life in which muscles weaken over time and once-automatic actions now require planning, assistance, or have become impossible.
Duchenne is a progressive genetic disorder, and people with the condition are not expected to live much beyond their mid-twenties. The author writes that even sleeping can be difficult and that bed sores have become an issue. He says he knows what the statistics say but is not ready to let them write the ending of his story. He wants to grow his career, get married, start a family, and wake up decades from now to realize that turning 25 was not the beginning of the end but simply another birthday.
For most Americans, he notes, an FDA commissioner is just another name in the news. For the roughly 30 million Americans living with a rare disease, that person can determine how many treatment options are available and how much time patients have to use them. He says he is not asking the agency to abandon science and respects doctors, clinical trials, and hard evidence. But he argues that Washington does not ask often enough who gets to decide how much risk a patient is willing to accept when a doctor and patient both understand the risks and there is real evidence a treatment could help.
Congress recognized part of that principle when President Trump signed the Right to Try Act into law in 2018, he writes. The law affirmed that 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. He describes Right to Try not as the end of the idea but as the beginning of a patient-centered philosophy that should shape the FDA's culture more broadly, including how the agency weighs evidence, risk, urgency, and access for rare-disease patients.
Senator Ron Johnson and Representative Diana Harshbarger have introduced Right to Try 2.0 to carry that principle into the next era of medicine, including individualized treatments that do not fit neatly into the FDA's traditional approval model. The author says they are right to press the issue, but adds that the next FDA leader should not have to wait for Congress to force a more patient-centered approach. He points to the agency's accelerated approval pathway, which exists because making patients with serious, life-threatening diseases wait years for a traditional clinical endpoint can cost them the thing they cannot get back. The pathway has already been used for multiple Duchenne treatments.
Waiting is never neutral when you have Duchenne, he writes. Every year can mean another loss of strength, another limitation, another piece of independence gone for good. His brother, who also lives with DMD, has seen the other side of the fight: even when a treatment exists, getting it can be its own battle. Traveling hours from home for a multiyear clinical trial sounds manageable in a Washington conference room, he says, but looks very different when you are the one making that trip while your body is working against you, rather than having access to the therapy at your own hospital.
The author says living with Duchenne has forced him to learn more about drug development and regulatory pathways than he ever expected. He wants an FDA that works with patients, physicians, and rare-disease experts. He also makes a personal appeal: he hopes whoever leads the agency brings on Dr. Houman Hemmati as deputy commissioner. Hemmati has spent his career in biotech rather than politics, building treatments for rare and degenerative diseases instead of regulating them from the outside. The author acknowledges that critics will say he is trying to get a friend into the room. He says they are right that he knows Hemmati, but they are missing that Hemmati does not just know his disease — he has lived it with him.
He is not asking anyone to promise him forever. He is asking for the chance to fight for the future he still sees. He has already outlived what many people expected, and he is not ready to stop. Senators considering Dr. Overton's nomination have a choice, he writes: confirm a commissioner who will treat patients as partners in their own survival, or keep a system that treats them as bystanders to it. It is time, he says, for Washington to understand what his life is worth.