I’m a mother and full-time caregiver asking the Food & Drug Administration and Commissioner Martin Makary to see what happens when regulatory caution meets a child with a terminal diagnosis. My son Ryu has Duchenne muscular dystrophy and his day-to-day life, along with sky-high medical bills and crushing urgency, are what this piece is about. I want regulators to understand that families live with risk every hour and that doing nothing is its own deadly choice.
For over four decades I have watched this disease take family members and now threaten my child, so my perspective is forged by loss and constant care. We live on one income with medical costs that would break most families, and the math is brutal when monthly treatments and supportive gear add up. Saying this plainly: we cannot wait while approval timelines move at the pace of paperwork.
Back when Dr. Makary stepped into his role he spoke about removing barriers and acting with flexibility, and that promise gave families real hope. Instead, therapies have stalled in long reviews, accelerated pathways have been overlooked, and some previously available options were effectively shelved. That gap between promise and practice is not an abstract policy debate for us; it determines whether a child breathes unaided tomorrow.
I watch the muscles in my son’s body tighten and fail, and I live with the small, heartbreaking losses that add up to a future narrowed by disease. Nights require a machine to help him breathe and days demand braces and constant help to manage hips and feet that no longer cooperate. He still grins and jokes, but the clock is always ticking and the decisions made in Washington decide how many more sunsets he gets.
I’M A PHYSICIAN AND I’M WORRIED THAT OUR HEALTH AGENCIES ARE FACING INCREASING CHAOS is a stark sentence some use to describe federal agencies, and from my household that chaos looks like hope eroding. The rare disease community initially believed the FDA would prioritize patients, but our experience has been exactly the opposite. When regulatory leaders hide behind caution we are the ones who pay the cost in irreversible decline.
The leadership of the FDA matters in concrete ways, and the departure of certain officials opens a real chance to change course. The center that reviews biologics has a lot of power over access to life-changing therapies, and who sits in that chair should understand that families are the final arbiters of acceptable risk. Appointing people who respect patient choice would be a simple, bold step toward rebuilding trust.
LOUISIANA SOCIAL WORKER FIGHTS STATE RED TAPE BLOCKING HER FROM HELPING SPECIAL NEEDS KIDS reads like more proof that bureaucracy often chokes compassion. Red tape at the state or federal level too frequently sidelines people trying to help, and that includes clinicians and advocates who want better access to treatments. If bureaucracy keeps good options from patients, it needs to be fixed, not defended.
Risk is not theoretical for families like mine; it is daily reality and careful calculation. We are not asking for reckless abandon of science or standards, but for recognition that paralysis is itself a deadly policy. Regulatory timelines must match the urgency of terminal illnesses, not the other way around.
DR. MEHMET OZ, STEPHANIE CARLTON: CALIFORNIA WAS HORRIBLY WRONG ABOUT GENDER AND KIDS appears in headlines for other culture fights, and it reminds me that political decisions have human consequences across many issues. For rare disease families those consequences are immediate and brutal when access is delayed or blocked. Leaders who want public trust must show they will fight for patients, not hide behind process.
Families like mine are asking for inclusion and transparency in decision making, and for processes that let parents participate early and meaningfully. Invite regulators into living rooms, not just hearing rooms, so they see the real stakes and faces tied to every dataset. That kind of accountability would change how risk is balanced and could save lives, which should be the point of any agency charged with public health.
My invitation to Commissioner Makary is simple: come meet Ryu and sit where choices become real. Let this be more than a photo op; let it be a reminder that every delayed approval costs a family more than paperwork can measure. For us, there are no do-overs and we need decisions that reflect that urgency now.
