ELEVIDYS voices
From diagnosis to treatment to the days beyond, life with Duchenne is a journey. Here you can explore stories from others in the community about ELEVIDYS, find comfort in shared experiences, and discover different families’ perceptions in their own words.
The stories featured here include families with younger children, since treatment was initially approved for a limited age range. Now that ELEVIDYS is approved for people 4 years of age and older, we’ll be sharing more stories from the broader community. Check back often for updates!
Reach out to us if you’d like to share your story with the community.
A GROWING COMMUNITY
From diagnosis to treatment to the days beyond, life with Duchenne is a journey. Here, you can explore stories from some of the more than 1,000 families who, together with their doctors, chose ELEVIDYS.*
Families have been compensated for the time required to share their story.
*Includes worldwide clinical trial and US commercial use through November 2025.
Charlie (shown here at age 8)
“Make a commitment to learning everything that you can about the treatment that you're looking at, whether it be gene therapy or something else.”
-Cheryl, Charlie's mom
Andrew | Shown here at age 5
"On treatment day, I thought of my brother, and I felt so grateful for this medicine."
- Jessica, Andrew's mom
Jason | Shown here at age 6
“The entire team at the hospital was so helpful. We were comfortable because we knew there was going to be someone there with us every step of the way.”
- Sylvia, Jason’s mom
Jaxson | Shown here at age 6
“After learning about ELEVIDYS, I knew I wanted Jaxson to have this opportunity.”
- Angela, Jaxson’s mom
Julius | Shown here at age 6
“Looking back at all the circumstances that led us to ELEVIDYS and his treatment, there’s no doubt that we would make the same decision over again.”
- Matthew, Julius’ dad
Nathaniel | Shown here at age 6
“Having treatment with ELEVIDYS was not an easy decision — I was a nervous wreck! — but it was the right choice for Nathaniel.”
- Carolyn, Nathaniel’s mom
“We spent a long time discussing treatment with doctors and our medical staff, which helped us feel comfortable that this would be the right choice for Max.”
Tao, Max's dad
Alex | Shown here at age 6
“Making the decision to move forward was the hardest of my life. After educating myself and discussing it with family, I trusted my gut … every day I’m grateful I did.”
- Daylee, Alex’s mom
Carter | Shown here at age 4
“I had to become a lion for my son.”
- Kristen, Carter’s mom
Jaxson, shown here at age 6
ELEVIDYS through a different lens
My sweet boy Jaxson has autism and faces significant cognitive challenges that have impacted his Duchenne journey and his ELEVIDYS treatment.
It breaks my heart to see him struggle. But in some ways, it brings a sense of peace to think that his way of seeing the world may protect him from fully feeling the weight of Duchenne’s challenges. Instead, Jaxson gets to focus on doing the things that really matter to him, like dressing up as a superhero or playing with his dinosaurs.
After learning about ELEVIDYS, I knew I wanted Jaxson to have this opportunity.
On treatment day, I explained that we were going to the clinic for medicine to help his muscles. He didn’t think much of it, because he is used to going to doctors’ appointments. But I still worried he would get anxious. I let him wear his favorite pajamas and brought his cherished teddy bear and blanket.
When we arrived at the clinic, he started getting nervous and clung to me like a spider monkey. Unlike some other children with autism, Jaxson loves being held, so I climbed into bed with him and stayed by his side. The clinic’s child development specialists brought him light-up toys and dinosaurs.
Looking back, I realize I didn’t need to be so nervous. Jaxson handled it much better than I expected.
To other families with a neurodiverse child, my advice is to trust your instincts on how to make them comfortable during their ELEVIDYS treatment. Rely on the deep connection you share to guide you. You know your child best.
Angela, Jaxson's mom
This story is a summary of one person’s experience and does not include all relevant information about ELEVIDYS. Please review the ELEVIDYS Treatment Guide for important information, including eligibility, safety, and monitoring.
Please see Important Safety Information and full Prescribing Information, including Boxed Warning, and Medication Guide for ELEVIDYS.
01/26 C-GT01-US-0474-V1
Jaxson, shown here at age 6
ELEVIDYS through a different lens
My sweet boy Jaxson has autism and faces significant cognitive challenges that have impacted his Duchenne journey and his ELEVIDYS treatment.
It breaks my heart to see him struggle. But in some ways, it brings a sense of peace to think that his way of seeing the world may protect him from fully feeling the weight of Duchenne’s challenges. Instead, Jaxson gets to focus on doing the things that really matter to him, like dressing up as a superhero or playing with his dinosaurs.
After learning about ELEVIDYS, I knew I wanted Jaxson to have this opportunity.
On treatment day, I explained that we were going to the clinic for medicine to help his muscles. He didn’t think much of it, because he is used to going to doctors’ appointments. But I still worried he would get anxious. I let him wear his favorite pajamas and brought his cherished teddy bear and blanket.
When we arrived at the clinic, he started getting nervous and clung to me like a spider monkey. Unlike some other children with autism, Jaxson loves being held, so I climbed into bed with him and stayed by his side. The clinic’s child development specialists brought him light-up toys and dinosaurs.
Looking back, I realize I didn’t need to be so nervous. Jaxson handled it much better than I expected.
To other families with a neurodiverse child, my advice is to trust your instincts on how to make them comfortable during their ELEVIDYS treatment. Rely on the deep connection you share to guide you. You know your child best.
Angela, Jaxson's mom
This story is a summary of one person’s experience and does not include all relevant information about ELEVIDYS. Please review the ELEVIDYS Treatment Guide for important information, including eligibility, safety, and monitoring.
Please see Important Safety Information and full Prescribing Information, including Boxed Warning, and Medication Guide for ELEVIDYS.
01/26 C-GT01-US-0474-V1
Nathaniel, shown here at age 6
Finding light in the dark
When we heard the news of Nathaniel’s diagnosis, it was as if the world went dark. The more we learned about Duchenne, the more overwhelmed and alone we felt.
A glimmer of light appeared when our doctor shared that the FDA had approved the first gene therapy for Duchenne. There was a lot to consider as he walked us through the treatment process, but we felt so supported knowing Nathaniel would be closely monitored and our doctor would be there to answer questions. Our faith gave us additional comfort when making this difficult choice. Even our SareptAssist contact was by our side the entire time, checking in to see how we were doing and answering our many questions.
Having treatment with ELEVIDYS is not an easy decision, but it was the right choice for Nathaniel.
On treatment day, I was a nervous wreck the entire 2-hour drive to the treatment center. I knew in my heart this was the right decision, but I was scared that something would go wrong. However, when we arrived, everyone was so kind and supportive. Nathaniel was anxious about getting poked with the IV needle, but his care team gave him a teddy bear and let him put a little IV in it. Once the infusion started, the nurses brought him toys and he watched TV. We couldn’t have asked for a better care team.
Walking this path can feel daunting, but know there are others here to help you find your way. I am infinitely thankful for the many people who were with us at every step!
Carolyn, Nathaniel's mom
This story is a summary of one person’s experience and does not include all relevant information about ELEVIDYS. Please review the ELEVIDYS Treatment Guide for important information, including eligibility, safety, and monitoring.
Please see Important Safety Information , full Prescribing Information , including Boxed Warning, and Medication Guide for ELEVIDYS.
01/26 C-GT01-US-0508-V1
Nathaniel, shown here at age 6
Finding light in the dark
When we heard the news of Nathaniel’s diagnosis, it was as if the world went dark. The more we learned about Duchenne, the more overwhelmed and alone we felt.
A glimmer of light appeared when our doctor shared that the FDA had approved the first gene therapy for Duchenne. There was a lot to consider as he walked us through the treatment process, but we felt so supported knowing Nathaniel would be closely monitored and our doctor would be there to answer questions. Our faith gave us additional comfort when making this difficult choice. Even our SareptAssist contact was by our side the entire time, checking in to see how we were doing and answering our many questions.
Having treatment with ELEVIDYS is not an easy decision, but it was the right choice for Nathaniel.
On treatment day, I was a nervous wreck the entire 2-hour drive to the treatment center. I knew in my heart this was the right decision, but I was scared that something would go wrong. However, when we arrived, everyone was so kind and supportive. Nathaniel was anxious about getting poked with the IV needle, but his care team gave him a teddy bear and let him put a little IV in it. Once the infusion started, the nurses brought him toys and he watched TV. We couldn’t have asked for a better care team.
Walking this path can feel daunting, but know there are others here to help you find your way. I am infinitely thankful for the many people who were with us at every step!
Carolyn, Nathaniel's mom
This story is a summary of one person’s experience and does not include all relevant information about ELEVIDYS. Please review the ELEVIDYS Treatment Guide for important information, including eligibility, safety, and monitoring.
Please see Important Safety Information , full Prescribing Information , including Boxed Warning, and Medication Guide for ELEVIDYS.
01/26 C-GT01-US-0508-V1
Nathaniel, se muestra aquí a los 6 años
Encontrar luz en la oscuridad
Cuando nos enteramos del deagnóstico de Nathaniel, fue como si el mundo se oscureciera. Cuanto más conocíamos sobre la enfermedad de Duchenne, más abrumados y solos nos sentíamos.
Un rayo de luz apareció cuando nuestro médico dijo que la Administración de Alimentos y Medicamentos (Food and Drug Administration, FDA) había aprobado la primera terapia genética para Duchenne. Había mucho por considerar mientras nos explicaba el proceso de tratamiento, pero nos sentimos muy apoyados al saber que Nathaniel sería supervisado de cerca y que nuestro médico estaría allí para responder nuestras preguntas. Nuestra fe nos dio más tranquilidad a la hora de tomar esta decisión difícil. Incluso nuestro contacto de SareptAssist estuvo a nuestro lado todo el tiempo, comunicándose para ver cómo estábamos y respondiendo nuestras muchas preguntas.
Recibir tratamiento con ELEVIDYS no es una decisión fácil, pero fue la elección correcta para Nathaniel.
El día del tratamiento, estuve nerviosa durante las 2 horas que duró el trayecto hasta el centro de tratamiento. En mi corazón sabía que esta era la decisión correcta, pero tenía miedo de que algo saliera mal. Sin embargo, cuando llegamos, todos fueron muy amables y comprensivos. A Nathaniel le daba miedo que lo pincharan con la aguja intravenosa, pero su equipo de asistencia le dio un osito de peluche y dejó que le pusiera una vía intravenosa pequeña. Una vez que comenzó la infusión, el personal de enfermería le llevó juguetes y se puso a ver televisión. No pudimos haber tenido un mejor equipo de asistencia.
Recorrer este camino puede parecer abrumador, pero debe saber que aquí hay otros para ayudarlo a encontrar su camino. Estoy infinitamente agradecida por las muchas personas que nos acompañaron en cada paso.
Carolyn, mamá de Nathaniel
Esta historia es un resumen de la experiencia de una persona y no incluye toda la información relevante sobre ELEVIDYS. Revise la Guía de tratamiento de ELEVIDYS para obtener información importante, incluida la elegibilidad, la seguridad y la supervisión.
Consulte la Información importante de seguridad y la Información de prescripción completa de ELEVIDYS.
Nathaniel, se muestra aquí a los 6 años
Encontrar luz en la oscuridad
Cuando nos enteramos del deagnóstico de Nathaniel, fue como si el mundo se oscureciera. Cuanto más conocíamos sobre la enfermedad de Duchenne, más abrumados y solos nos sentíamos.
Un rayo de luz apareció cuando nuestro médico dijo que la Administración de Alimentos y Medicamentos (Food and Drug Administration, FDA) había aprobado la primera terapia genética para Duchenne. Había mucho por considerar mientras nos explicaba el proceso de tratamiento, pero nos sentimos muy apoyados al saber que Nathaniel sería supervisado de cerca y que nuestro médico estaría allí para responder nuestras preguntas. Nuestra fe nos dio más tranquilidad a la hora de tomar esta decisión difícil. Incluso nuestro contacto de SareptAssist estuvo a nuestro lado todo el tiempo, comunicándose para ver cómo estábamos y respondiendo nuestras muchas preguntas.
Recibir tratamiento con ELEVIDYS no es una decisión fácil, pero fue la elección correcta para Nathaniel.
El día del tratamiento, estuve nerviosa durante las 2 horas que duró el trayecto hasta el centro de tratamiento. En mi corazón sabía que esta era la decisión correcta, pero tenía miedo de que algo saliera mal. Sin embargo, cuando llegamos, todos fueron muy amables y comprensivos. A Nathaniel le daba miedo que lo pincharan con la aguja intravenosa, pero su equipo de asistencia le dio un osito de peluche y dejó que le pusiera una vía intravenosa pequeña. Una vez que comenzó la infusión, el personal de enfermería le llevó juguetes y se puso a ver televisión. No pudimos haber tenido un mejor equipo de asistencia.
Recorrer este camino puede parecer abrumador, pero debe saber que aquí hay otros para ayudarlo a encontrar su camino. Estoy infinitamente agradecida por las muchas personas que nos acompañaron en cada paso.
Carolyn, mamá de Nathaniel
Esta historia es un resumen de la experiencia de una persona y no incluye toda la información relevante sobre ELEVIDYS. Revise la Guía de tratamiento de ELEVIDYS para obtener información importante, incluida la elegibilidad, la seguridad y la supervisión.
Consulte la Información importante de seguridad y la Información de prescripción completa de ELEVIDYS.
Alex, se muestra aquí a los 6 años
Una elección reflexiva
Estoy muy contenta de haber elegido ELEVIDYS para Alex; sin embargo, tomar la decisión fue lo más difícil de mi vida. Cuando su médico le recomendó ELEVIDYS por primera vez, fue difícil saber si era el camino correcto para nosotros. Alex tenía solo 5 años y me preocupaba correr un riesgo con mi dulce hijito, pero mi instinto me decía que necesitaba aprender más.
Investigué durante horas y lo hablé muchas veces con el médico de Alex. En una cita, el médico me ayudó a considerar las ventajas y desventajas, y respondió todas las preguntas a las que no tenía respuesta. La opinión de mi familia también fue importante porque siempre fueron mi mayor sistema de apoyo para Alex. Estuvieron de acuerdo con el papá de Alex, que había dicho: “Si no hacemos esto, siempre nos preguntaremos lo que podría haber sido”. Aunque estaba preocupada, como madre, tuve que seguir mi instinto y seguir adelante con el tratamiento.
Después de informarme y hablarlo con mi familia, confié en mi instinto y elegí ELEVIDYS.
Cuando llegó el día del tratamiento, estaba muy nerviosa. Respiré hondo muchas veces, abracé mucho a Alex y confié en mí misma.
Hoy, 9 meses después del tratamiento, estoy muy contenta con el camino que elegí y agradecida con las personas que me animaron a pensar en todas las posibilidades. Elegir ELEVIDYS no fue fácil, pero todos los días estoy agradecida de haberlo hecho.
Esta historia es un resumen de la experiencia de una persona y no incluye toda la información relevante sobre ELEVIDYS. Revise la Guía de tratamiento de ELEVIDYS para obtener información importante, incluida la elegibilidad, la seguridad y la supervisión.
Consulte la Información importante de seguridad y la Información de prescripción completa de ELEVIDYS.
Alex, se muestra aquí a los 6 años
Una elección reflexiva
Estoy muy contenta de haber elegido ELEVIDYS para Alex; sin embargo, tomar la decisión fue lo más difícil de mi vida. Cuando su médico le recomendó ELEVIDYS por primera vez, fue difícil saber si era el camino correcto para nosotros. Alex tenía solo 5 años y me preocupaba correr un riesgo con mi dulce hijito, pero mi instinto me decía que necesitaba aprender más.
Investigué durante horas y lo hablé muchas veces con el médico de Alex. En una cita, el médico me ayudó a considerar las ventajas y desventajas, y respondió todas las preguntas a las que no tenía respuesta. La opinión de mi familia también fue importante porque siempre fueron mi mayor sistema de apoyo para Alex. Estuvieron de acuerdo con el papá de Alex, que había dicho: “Si no hacemos esto, siempre nos preguntaremos lo que podría haber sido”. Aunque estaba preocupada, como madre, tuve que seguir mi instinto y seguir adelante con el tratamiento.
Después de informarme y hablarlo con mi familia, confié en mi instinto y elegí ELEVIDYS.
Cuando llegó el día del tratamiento, estaba muy nerviosa. Respiré hondo muchas veces, abracé mucho a Alex y confié en mí misma.
Hoy, 9 meses después del tratamiento, estoy muy contenta con el camino que elegí y agradecida con las personas que me animaron a pensar en todas las posibilidades. Elegir ELEVIDYS no fue fácil, pero todos los días estoy agradecida de haberlo hecho.
Esta historia es un resumen de la experiencia de una persona y no incluye toda la información relevante sobre ELEVIDYS. Revise la Guía de tratamiento de ELEVIDYS para obtener información importante, incluida la elegibilidad, la seguridad y la supervisión.
Consulte la Información importante de seguridad y la Información de prescripción completa de ELEVIDYS.
Alex, shown here at age 6
A thoughtful choice
I’m so happy I chose ELEVIDYS for Alex; however, making the decision was the hardest of my life. When his doctor first recommended ELEVIDYS, it was difficult to know if it was the right path for us. Alex was only 5 years old, and I was worried I’d be taking a risk with my sweet little boy, but my gut was telling me I needed to learn more.
I did hours of research and talked it through with Alex’s doctor many times. At one appointment, she helped me weigh the pros and cons and answered all my questions I did not have answers to. My family’s opinion was also important because they have always been my biggest support system for Alex. They agreed with Alex’s dad, who had said, “If we don’t do this, we will always wonder what could have been.” Even though I was worried, as a mom, I had to follow my gut and move forward with treatment.
After educating myself and discussing it with family, I trusted my gut and chose ELEVIDYS.
Today, 9 months after treatment, I am so happy with the path I chose, and grateful to the people who encouraged me to think through all the possibilities. Choosing ELEVIDYS was not easy, but every day I am grateful I did.
Daylee, Alex’s mom
This story is a summary of one person’s experience and does not include all relevant information about ELEVIDYS. Please review the ELEVIDYS Treatment Guide for important information, including eligibility, safety, and monitoring.
Please see Important Safety Information , full Prescribing Information , including Boxed Warning, and Medication Guide for ELEVIDYS.
01/26 C-GT01-US-0509-V1
Alex, shown here at age 6
A thoughtful choice
I’m so happy I chose ELEVIDYS for Alex; however, making the decision was the hardest of my life. When his doctor first recommended ELEVIDYS, it was difficult to know if it was the right path for us. Alex was only 5 years old, and I was worried I’d be taking a risk with my sweet little boy, but my gut was telling me I needed to learn more.
I did hours of research and talked it through with Alex’s doctor many times. At one appointment, she helped me weigh the pros and cons and answered all my questions I did not have answers to. My family’s opinion was also important because they have always been my biggest support system for Alex. They agreed with Alex’s dad, who had said, “If we don’t do this, we will always wonder what could have been.” Even though I was worried, as a mom, I had to follow my gut and move forward with treatment.
After educating myself and discussing it with family, I trusted my gut and chose ELEVIDYS.
Today, 9 months after treatment, I am so happy with the path I chose, and grateful to the people who encouraged me to think through all the possibilities. Choosing ELEVIDYS was not easy, but every day I am grateful I did.
Daylee, Alex’s mom
This story is a summary of one person’s experience and does not include all relevant information about ELEVIDYS. Please review the ELEVIDYS Treatment Guide for important information, including eligibility, safety, and monitoring.
Please see Important Safety Information , full Prescribing Information , including Boxed Warning, and Medication Guide for ELEVIDYS.
01/26 C-GT01-US-0509-V1
Andrew, shown here at age 5
Decisions rooted in our story
I tried so hard not to cry when my son Andrew was diagnosed with Duchenne. My brother had passed away from Duchenne 22 years earlier, so I knew what Andrew’s future might hold. But then the doctor said something I’ll never forget: “Andrew’s journey will be different than your brother’s. There are treatments that were not available to your brother, and there are clinical trials under way.” Those words gave me hope.
About a year later, my husband and I felt so grateful when our doctor called to tell us that ELEVIDYS had been approved and that Andrew qualified for treatment.
Duchenne prevents Andrew’s body from making dystrophin, which causes damage to his muscles over time. We understood there were potential risks with ELEVIDYS and talked through them with our doctor. So, after talking about it and praying about it, my husband and I felt it was the right choice for us to have Andrew treated with ELEVIDYS as soon as possible.
On treatment day, I thought of my brother, and I felt so grateful for this medicine.
As I reflect on Andrew’s treatment a year and a half later, I cry tears of happiness when I watch my confident boy going for walks with our family or playing in the playground. He told me recently, “I’m going to be brave and go down the slide!” I love being able to experience that with him. Seeing Andrew’s joy — and remembering my brother — I feel grateful for choosing ELEVIDYS.
Jessica, Andrew's mom
This story is a summary of one person’s experience and does not include all relevant information about ELEVIDYS. Please review the ELEVIDYS Treatment Guide for important information, including eligibility, safety, and monitoring.
Please see Important Safety Information and full Prescribing Information, including Boxed Warning, and Medication Guide for ELEVIDYS.
01/26 C-GT01-US-0474-V1
Andrew, shown here at age 5
Decisions rooted in our story
I tried so hard not to cry when my son Andrew was diagnosed with Duchenne. My brother had passed away from Duchenne 22 years earlier, so I knew what Andrew’s future might hold. But then the doctor said something I’ll never forget: “Andrew’s journey will be different than your brother’s. There are treatments that were not available to your brother, and there are clinical trials under way.” Those words gave me hope.
About a year later, my husband and I felt so grateful when our doctor called to tell us that ELEVIDYS had been approved and that Andrew qualified for treatment.
Duchenne prevents Andrew’s body from making dystrophin, which causes damage to his muscles over time. We understood there were potential risks with ELEVIDYS and talked through them with our doctor. So, after talking about it and praying about it, my husband and I felt it was the right choice for us to have Andrew treated with ELEVIDYS as soon as possible.
On treatment day, I thought of my brother, and I felt so grateful for this medicine.
As I reflect on Andrew’s treatment a year and a half later, I cry tears of happiness when I watch my confident boy going for walks with our family or playing in the playground. He told me recently, “I’m going to be brave and go down the slide!” I love being able to experience that with him. Seeing Andrew’s joy — and remembering my brother — I feel grateful for choosing ELEVIDYS.
Jessica, Andrew's mom
This story is a summary of one person’s experience and does not include all relevant information about ELEVIDYS. Please review the ELEVIDYS Treatment Guide for important information, including eligibility, safety, and monitoring.
Please see Important Safety Information and full Prescribing Information, including Boxed Warning, and Medication Guide for ELEVIDYS.
01/26 C-GT01-US-0474-V1