Yesterday, I had the opportunity to be at the FDA and attend the Advisory Committee meeting discussing Deramiocel, an investigational therapy aimed at supporting cardiomyopathy and upper limb function in Duchenne muscular dystrophy for patients like my son, Bennett.
I’m someone who often needs time to process an experience before I can truly put it into words.
After sitting with it, here are my thoughts. First, I am grateful I was able to be there. As a mother of a boy who is still relatively young and doing quite well for someone living with Duchenne, it was an emotional and overwhelming day.
There were countless slides, charts, and years of data to absorb. But what affected me most wasn’t the data—it was the people. I found myself in tears multiple times listening to mothers, patients, and advocates share their stories, their experiences, and their truths.
Many of them were strangers to me. Some I still haven’t had the chance to meet personally. Yet I sat there in awe of them. One conversation that will stay with me was with the mother of a 24-year-old young man with Duchenne. This therapy has helped him and, because of his specific mutation, it has essentially been his only option throughout his journey. I could see how nervous she was before speaking, but I could also see how desperately important it was for her story to be heard. Really heard and for her son to be seen.
I have to admit that I left feeling disappointed. I was disappointed that the FDA called this meeting and three voting committee members didn’t even attend. I was disappointed by the back-and-forth that at times felt more argumentative than productive, and by questions that were seemingly unanswered. Most of all, I was disappointed that while the patient and caregiver testimonies were acknowledged, they still felt overlooked. I understand there are regulations. I understand there are standards. Of course, there has to be. And maybe the data was, as they repeatedly said, “too fragile.”But shouldn’t some of that decision belong to us? As the mother of a six-year-old boy with Duchenne, I can tell you with certainty that I am listening to parents. I am listening to lived experience. I am listening to the people who wake up every day and navigate this disease. Data can be fragile, but families deserve the right to weigh the risks and make informed decisions for themselves.
One of the adult advocates who spoke on his own behalf challenged everyone in the room to imagine what it is like to need help using the bathroom. To have a sandwich sitting in front of you when you’re starving and be unable to pick it up. To have an itch driving you crazy but you have to wait for someone else to scratch it. Can you imagine it?Most people can’t. Most people will never have to. But those living with Duchenne don’t have that luxury, and they don’t have that time.
While I was disappointed by the 9–3 vote against recommending approval, I was not disappointed in our community. In fact, I continue to be amazed by it. This is a community I never wanted to be part of and never imagined I would be. Yet I am endlessly grateful for the people it has brought into my life. The work these advocates have done—long before my family’s journey even began—is incredible beyond belief.
One thing about the Duchenne community: we will always show up.It was wonderful to meet more parents and patients, even if my trip was too short because of travel delays. I only wish I could have met more of you. And as always, it was so special to spend time with friends I’ve made over the past three years. These are friendships that will last a lifetime because no one truly understands this journey unless they’re living it.
The FDA has until August 22, 2026, to make its decision.
My hope is that they truly heard what our community had to say. These patients deserve dignity. And I refuse to let my son lose his.
