Every Duchenne journey is different. Here, people living with Duchenne and others connected to the community share their experiences in their own words. As the community reflects on the 10 years since the U.S. FDA approval of the first therapy specifically for Duchenne, they look back on a decade of evolving care and changing possibilities.
Whether you're interested in Krishna's experience balancing college life with competitive power soccer, Alpa's perspective as a mother whose family has experienced firsthand how Duchenne care has changed over time, Darrel's journey as a content creator and storyteller, Aditya's experience learning to advocate for himself while building a career in aerospace technology or Kara's perspective from the front lines of Duchenne care, each story offers a different view of what the past decade has meant to people across the Duchenne community.
Krishna Khushalani
18 years old
Living with Duchenne
College Student, Power Soccer Athlete
"When I was younger, I never thought I'd be playing a sport in college. Now I'm at Auburn University studying aerospace engineering and playing wheelchair soccer as a scholarship student-athlete.
Power soccer was one of the biggest reasons I chose Auburn. They have one of the best club teams. I practice three times a week, and playing at this level is intense. But I feed off the energy of the competition. The pressure is high, but that's part of what makes it exciting.
Sports have taught me a lot about myself. I've always had a drive to be successful and to do things people might say are impossible. When I'm on the court, I'm not limited by my disability. I can do things that everyone else does.
The transition to college has been great and I like my classes. I grew up fascinated by airplanes and rockets, and my goal is to design aircraft or work in a rocket lab one day. I'm also interested in making air travel more accessible. There has to be a better way to help people with disabilities travel more independently, and I'd love to be part of finding it.
I feel very lucky. I know that a lot of guys my age aren't able to do some of things that I can still do independently. A lot of things have gone the right way with my care team, and I'm grateful to be where I am. I’ve been getting weekly infusions of exon-skipping therapy for more than 9 years. I live on campus on my own, and a nurse comes to my dorm room once a week. Everyone has things they have to do. That’s the same for me. For two hours a week, I have to get an infusion. It's just part of my life.
I know younger kids with Duchenne are watching. I try to be positive and show them what's possible. Whether it's going to college, playing sports or living independently, there's still so much you can do. You might be limited in some ways, but there are a lot of things you can do, and I want them to see that for themselves.”
Alpa Khushalani
Duchenne Mom and Advocate
"When Krishna was born, he had a hole in his heart and clubfoot. For years, we thought many of the challenges we were seeing were connected to those conditions. It wasn't until he was almost 6 that a neurologist watched him stand up and said, ‘I think your son has Duchenne.’ It took us six years to get there, and it took that neurologist about six seconds. I remember thinking, I thought his health issues were behind us.
At that time, there were no approved therapies. We didn't even have an ICD-10 code for Duchenne. Muscular dystrophies were lumped together. Our only option was to put him on steroids and wait.
Then, when we learned Krishna’s mutation, his doctor told us exon-skipping therapies were showing a lot of potential. When the opportunity came to participate in a clinical trial for a Sarepta therapy, we were so happy because – at that point – it was the trial or nothing. It was hard not knowing if we could receive placebo or therapy. But we had seen data that looked encouraging, so we decided to take the chance. You trust the science and that God has a plan and do everything you can for your child.
I talk with newly diagnosed families all the time, and the conversations are very different from the ones we were having when Krishna was diagnosed. We're now talking about approved therapies, newborn screening and getting treatments to children earlier. Krishna is 18 and living independently at college. He's studying aerospace engineering, playing power soccer and planning for his future.
The thing that brings me the most joy today is watching Krishna play soccer. As a parent, I watched my other boys play sports growing up, and I wasn't sure Krishna would ever have that experience. Now I get to see that big smile on his face, his confidence, his teammates and his friendships. It makes my heart happy.
This was not what we thought the trajectory of Duchenne would look like. We feel very fortunate.”
Darrel Nicklow, Jr.
28 years old
Living with Duchenne
Content Creator, Graphic Designer
“I was diagnosed with Duchenne when I was 5. I had a heart transplant when I was 13. And I’ve been receiving weekly infusions of exon-skipping therapy since 2017. Every Thursday, I have an infusion. It's just part of my life.
But I don’t talk about Duchenne very much. I don't want that to be the main thing about me. It would take away from who I am. I have a bachelor’s degree in Digital Media Design and a master’s degree in Educational Technology, with a minor in interdisciplinary studies. I started my own company and work as a graphic designer. I'm also a content creator and sports storyteller.
These days, I'm focused on being happy. I want to be healthy, I want to make people proud of me and I want to keep doing the things I love.
I've been a sports fan my entire life. In 2020, my mom gave me a camera and I started making videos. Now I travel to games, interview players and tell stories on my YouTube channel. I want people to see the atmosphere, the crowd, the personalities and what makes those athletes interesting. A lot of times, I'm telling stories about people and teams that don't get much recognition.
Every day I’m working. It fuels me. Most people would have a team helping them. I'm doing it all on my own.
These days, I'm focused on being happy. I want to be healthy, I want to make people proud of me and I want to keep doing the things I love.
It’s cool to think about that I was one of the early patients on exon-skipping therapy. It’s like I'm one of the founding fathers. Without this medicine, who knows if other medicines would have been developed. I helped create a chain reaction. Being part of that means a lot to me.”
Aditya Nair
25 years old
Living with Duchenne
Inflight Software Developer
“I was diagnosed with Duchenne at 6. I’m 25 now, on an exon-skipping therapy and have been steady for a couple of years.
Back when I was in high school and thinking about a future career, I looked for something that matched my strengths and interests. I got into computer science, and the more I learned, the more I realized it was a good fit. When I’m coding, I get what I call a ‘coder’s high.’ I don’t want to stop until I get to the solution, and I can stay at it for hours.
I write code that helps a spacecraft dock with a space station in lunar orbit. It’s a huge honor to use my degree in a way that feels meaningful and to contribute to something that could have a real impact.
I earned my degree in computer science, got a job and moved into my own condo. I also started working toward a master’s degree in AI.
Independence is really important to me. When I was in college, I started going to doctor’s appointments by myself. When my parents were with me, people didn’t always speak directly to me. Going on my own gave me the chance to ask questions and speak for myself. That’s when I got into the habit of being my own advocate.
I think that’s becoming more important for people growing up with Duchenne. As new treatments and advances continue to change what’s possible, more people are thinking about college, careers and what comes next.
For me, the next frontier is space. I was always a space guy, and a huge Star Wars fan growing up. Now, I write code that helps a spacecraft dock with a space station in lunar orbit. The coolest part is playing my part, however small, in helping put American boots back on the Moon and knowing that the work my team is doing today could help pave the way for humans to go even farther in the future.
It’s a huge honor to use my degree in a way that feels meaningful and to contribute to something that could have a real impact.”
Kara Godwin, DNP, PNP
Pediatric neuromuscular nurse practitioner
Director, Medical Affairs, Sarepta
“The approval of the first treatment for Duchenne was an exciting and unforgettable time. I was a pediatric nurse practitioner working as a neuromuscular care coordinator at UF Health Shands Children’s Hospital at the time. Watching a treatment progress from clinical trial participation to becoming an option we could offer in clinic felt monumental, almost as if we were witnessing history unfold before our eyes.
Our team knew we would likely be the first to administer treatment commercially, and we were ready. In Duchenne, every minute represents muscle lost that may never be regained. I was determined not to let a single moment be wasted once treatment became available.
They taught me how to navigate conversations about risk and benefit in a space where there is no cure, but there are treatment options that aim to improve quality of life and alter the trajectory of disease.
So many moments from those years remain etched in my heart and mind. Through every interaction, one thing always stood out: the extraordinary resilience of patients and caregivers facing one of the most devastating diseases I have ever encountered. Duchenne affects far more than a person's ability to walk. It impacts every aspect of a patient's life and reaches every member of the family. Its progression is gradual but relentless, with each stage often bringing new challenges and difficult realities.
Those experiences strengthened my determination to translate scientific innovation into meaningful patient care. They taught me how to navigate conversations about risk and benefit in a space where there is no cure, but there are treatment options that aim to to improve quality of life and alter the trajectory of disease. They reinforced my commitment to do everything possible to advocate for what was best for each patient and family, recognizing that 'best' can look different for everyone.
The patients are still at the center of everything I do. They are who I think about every day, both those I continue to serve and those who remain in my memories. While my role has evolved, my purpose has not. I continue to advocate for the same goals, the same families, and the same hope for a better future, only now on a broader scale. Their resilience continues to inspire me, and their journeys remain the reason I do this work.”
Want to share your story?
For a decade, progress in Duchenne has been shaped by patients, families, clinicians and advocates. Your perspective is part of this community’s history and its future, and every experience matters. Below is an opportunity for you to share about your Duchenne journey.
Your story may appear on Sarepta’s website or other support materials. The personal information you provide makes it possible for us to help share your story and we will follow up with you if necessary.
