A virtual FDA meeting gave me the opportunity to advocate for my sons
For a busy mom and caregiver, attending in person isn't always an option
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The first thing I tell others about myself is that my husband and I share seven children: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Being a mom is literally my favorite thing about myself.
Caring for my family is my main purpose in life. Most mothers will tell you the same, but in addition to being a mom, I also serve as the primary caregiver to Max, Rowen, and Charlie, who all have Duchenne muscular dystrophy (DMD). My day, from the time my sons wake up until they go to bed, is theirs, and despite the many challenges involved, that is a great privilege.
This week was very important for the Duchenne community and gave me the opportunity to be an advocate as well as a mom and caregiver.
Sharing my sons’ experiences
On Wednesday, the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee met with Capricor Therapeutics to review the company’s resubmission of a Biologics License Application seeking approval of the cell therapy deramiocel for people with DMD.
Max, Rowen, and Charlie all participated in the Phase 3 clinical trial of deramiocel and have benefited from their involvement. Therefore, I felt compelled to lend my voice and share my sons’ experiences during the meeting’s open public hearing.
The decision to speak was easy. I believe in this therapy and have seen my sons maintain upper body strength and cardiac function, even in their late teens and early 20s, through their participation in the trial. I think the therapy should be available to everyone living with Duchenne.
When the FDA advisory committee meeting was first announced, it was scheduled to be virtual. This was ideal for me as a caregiver and a mom with a large family. However, it was later changed to an in-person meeting, with the option to join virtually.
Many members of the Duchenne community asked me if I would be there. Some wanted to meet in person, while others wanted to catch up, but most of all, it’s just nice to be together. No one pressured me to attend in person, but I still felt torn.
Traveling from the middle of Nebraska to Washington, D.C., is not easy, and it would have been impossible for me to travel alone with my three sons. It would have required several days away from home and my other children as well.
This summer has been tough for me. I’ve shared how Rowen’s broken leg, Max’s change in college status, looking for and purchasing a new house, and daily caregiving have left me teetering on the verge of burnout and exhaustion for several weeks. As nice as it would’ve been to connect with the Duchenne community face-to-face, I knew a last-minute trip to D.C. would have pushed me over the edge. I also worried that presenting virtually wouldn’t have the same impact.
I knew I didn’t want to go as soon as I found out the meeting would be held in person, but I felt guilty about it. So, instead of rushing to reply, I talked to my husband and my best Duchenne mom friend, spent a lot of time in prayer, and decided to follow my initial gut feeling.
Taking time to decide led me to the conclusion that my virtual presentation would still make an impact — and it did. I shared real-life moments and meaningful changes my sons have experienced because of deramiocel.
I also shared why I could not be there in person, and I think it gave the committee insight into life with a rare disease. You can’t just pick up and fly across the country on a week or two’s notice with three power wheelchairs, a Hoyer lift, and enough caregivers to cover the flights and hotel stays. It’s also not possible for a caregiver to just up and leave without the proper supports in place at home.
The virtual meeting option allowed me to be a caregiver, mom, and advocate all at once!
Note: Muscular Dystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.
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