My 14-year-old son Ryu has a terminal rare disease. We were to travel from Texas to the District of Columbia on May 12 to attend a Senate hearing on the Food and Drug Administration (FDA) budget. There, Ryu had hoped to meet our Senator, Ted Cruz, R-Texas, to shake his hand, and to tell him a little about what it’s like to live with Duchenne muscular dystrophy (DMD).
The hearing was canceled because then-FDA Commissioner Dr. Martin Makary left his post. The FDA continuing to block access to rare disease treatments remains a problem that he created, but President Donald Trump has the opportunity to change that by appointing a new FDA leader willing to listen to rare disease families and open treatment doors for my son and other adults and children with rare diseases.
Makary did neither.
DMD killed my beloved brothers Angelo and Antonio in their early twenties. It is a fatal genetic condition that progressively destroys muscles throughout the body. My son now faces my brothers’ fate.
In the 1990s, Angelo agreed to experimental spinal surgery to treat his DMD. Although he knew chances of survival were slim, he allowed doctors to put a metal rod in his spine, and thankfully, he lived several more years. Angelo did not hesitate, as he felt the treatment would help researchers understand more about this vicious disease.
That choice was his alone. No agency in far-off Washington, D.C., blocked him from taking his leap of faith and contributing to a possible cure.
Unlike when my brothers were alive, there are now therapies that can slow the progression of Duchenne and extend lives. Many patients are living higher-quality lives because of these advances.
Last summer, our family was looking into enrolling Ryu in a program for a DMD treatment called Elevidys. Today, we cannot even consider this treatment that could preserve Ryu’s muscle function, because the FDA has effectively made that decision for us by restricting treatment to ambulatory patients. They blocked the only gene therapy treatment for children who, like Ryu, are already in a wheelchair.
Makary once spoke about removing barriers and increasing regulatory flexibility for patients facing devastating illnesses, and for a moment, it seemed the system might work for us.
Instead, we saw increased delays made by bureaucrats who don’t deteriorate medically while we wait.
Today, Ryu’s arms are too weak to raise his hand to answer questions at school, even though he knows the answers. At night, he relies on breathing support because DMD has weakened the muscles that control his lungs.
Despite this, he still laughs, argues, and dreams about his lifelong goal of becoming a Navy SEAL.
Washington is full of government officials unaware of what Ryu and other rare disease patients go through every day. My job is to educate them, because their influence over FDA policy matters.
Last month, I invited Makary to my home to see firsthand what life is like for a child whose muscles are slowly wasting away. I now extend that invitation to acting FDA Commissioner Kyle Diamantas, and whomever the president permanently appoints to lead the agency.
As Ryu told me recently as he was using a machine to help him cough up mucus his lungs are too weak to handle, “Mom, this sucks. But you telling my story makes this a lot easier.”
The next FDA leaders have power over the life of my son. That’s why we wanted to come to Washington—to put a face to FDA treatment delays.
We are seeking partners from inside the halls of government—or as Ryu would call them, “heroes.”
It is up to the president and the U.S. Senate to ensure the next FDA appointee finally puts patients first.
Angelo chose faith over fear, and Ryu deserves that same choice. My brother made his decision for children he never even met, including the nephew born after he died. Can our government demonstrate the same resolve and courage as Angelo?
The FDA now has an opportunity to reset its relationship with rare disease families and help rather than obstruct. The new Commissioner must do the right thing and utilize existing protocols that accelerate treatment approvals for rare disease families and kids like Ryu. And listen.
It is too important an opportunity to squander.
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