My son has a terminal disease — why FDA delays are failing families like mine

Ive spent over 40 years on the front line against a terminal illness that killed my brothers and put my 14-year-old son into a wheelchair. This week, I asked Food & Drug Administration (FDA) Commissioner Dr. Martin Makary to leave his gilded office and come visit my home, because I fear that hes lost touch with terminally ill children like Ryu. 

Im Ryus full-time caretaker. Like most families, we rely on one income and have limited financial resources. What we do have are astronomical medical expenses; Ryus steroids alone are $30,000 per month. My husband and I thankfully found a nonprofit that covered the remaining $2,000 after our insurance paid their portion — but other expenses fall squarely on us. 

Its easy to ignore these struggles when youre a powerful official in Washington, D.C. In my house, Ryu struggles to breathe because of Duchenne muscular dystrophy, the same illness that I watched kill my brothers Angelo and Antonio at ages 20 and 22. Experimental drugs could let Ryu avoid their fate, but we cant access them under a regulatory environment that appears to prioritize bureaucratic caution over the lives of dying children. 

A year ago, the thought Dr. Makary would be our strongest ally. He promised to “remove barriers and exercise regulatory flexibility” to deliver meaningful treatments for patients. Families listened and dared to believe that the system would finally reflect the urgency of the conditions we face.

But our hopes were dashed over and over again as therapies were subjected to extended review cycles, , and previously authorized treatments were paused, restricted, or effectively sidelined.

The upcoming departure of FDAs Director of the Center for Biologics Evaluation and Research, Dr. Vinay Prasad, the man Makary assigned to make these life-and-death decisions, creates an opportunity for a reset. He oversaw the committee in charge of treatment approvals, so he is seen by many in the rare disease community as responsible for delaying treatments rather than delivering them.

Now, as Dr. Makary considers Dr. Prasads successor, he has a chance to appoint someone who understands that families should have the right to make their own decisions about which medicines are “risky” for their declining loved ones.

Risk is a big part of Ryus day-to-day, and our whole family is intimately acquainted with it. At night, he relies on a machine to keep his lungs functioning because they could fail at any time. He always dreamed of being a ; now he just hopes to live long enough to cast his first ballot.

Despite this, he is the happiest kid I know. And its important that Dr. Makary knows what that looks like, because when he and Dr. Prasad talk about risk, theyre doing so based on what the paperwork tells them. They make decisions that, from their offices, seem like they can be reviewed and revisited. 

But when I hear “risk,” I see Ryus quality of life irreversibly decline by the day. His calves are so tight that he cannot flatten his feet, so he wears special braces for some relief. During the day he wears a gait belt just to align his hips as his muscles deteriorate, and at night, he cannot even turn himself over without our help.

Under Ryus current treatment, these changes are irreversible. Thats why treatment options must be expanded — and not at the speed of the FDA, but at the speed of terminal rare diseases. 

Families understand risk better than anyone. We are not asking for reckless approvals or the abandonment of scientific standards. What we need is a system that recognizes that doing nothing is a decision that is deadly, 100% of the time.

If the FDA wants to rebuild trust, it needs to start with the families who have been burned. That means involving patient communities earlier in the process