For four decades, I’ve battled Duchenne muscular dystrophy. My brothers, Angelo and Antonio, lost their lives to this illness at ages 20 and 22. Antonio’s passing in 2015 was particularly hard, as my son, Ryu, was just an infant then and had already been diagnosed with the same heartbreaking condition.
When I think back to my childhood, I remember praying for my brothers and caring for them alongside my mother. We went to church nearly every day with all five of us kids in tow. I continually asked God to heal my brothers, and when Ryu was born, I included him in that prayer, too.
For more than 40 years, I’ve made the same plea for help and for my voice to be recognized.
But I had another request for God. I asked Him to give me a chance to share our family’s story, though I didn’t know how or when that would transpire. I truly believed it would happen.
This year, that prayer was heard. I was invited to advocate not just for my brother and son, but for all families facing the isolation of a terminal rare disease.
I traveled to Washington, D.C., where I told my story to lawmakers and patient advocates from both parties, urging them to push the Food and Drug Administration to support drugs like Erevidis, which is the only gene therapy available for my son’s illness.
Our advocacy was effective. I can’t gauge how much my small voice contributed, but the collective outcry certainly made a difference.
The first hint of success came when Dr. Vinay Prasad, director of the Center for Biologics Evaluation and Research, announced he was resigning from the FDA just a week later. He retired this month after blocking a potential life-saving treatment for children like my son across the nation.
I’m merely a mother, yet recently we celebrated Easter—a time when we remember how the carpenter saved the world. He challenged the systems of his time that sidelined the weak and sick. Even when he fell, he never backed down. I aspire to emulate that courage.
As Maundy Thursday approached again this year, Ryu was having a tough night. As he often does, he required a Bipap machine to assist his breathing. Despite the circumstances, he looked up at me and said, “Mom, this is tough, but what you’re doing is going to make it a lot easier.” My 14-year-old son, who’s in a wheelchair, is truly the happiest child I know.
My experiences might not resonate with FDA Director McCulley, but it feels like he’s forgotten Ryu and countless other children facing similar challenges. Yet, God sees us. He knows each of our stories even before we’re welcomed into the world. Sometimes, as Gabriel once told the prophet Daniel, our prayers are answered long before we see them realized.
For over 40 years, I’ve sent the same prayer for help and for my voice to be recognized. To the world, Antonio and Angelo may be distant memories, but they lay the foundation for how my husband and I care for Ryu. And God has allowed me to share their story, from El Paso to the nation’s heart.
Although Director Makari and Dr. Prasad may have lost sight of their mission to save lives, it seems God has a different plan. He’s just getting started in sharing the good news with me, and so far, it’s been extraordinary.
But I wasn’t astonished. I always believed that God would find a way.
