NS Pharma: We Speak Duchenne

Ambassador Brantley wearing a backpack in front of a teal background surrounded by fun iconography.

Duchenne
Stories

These are the inspiring stories of Duchenne Heroes. In their own words, find out how they stay motivated to push through their challenges and live life normally.

Personal Stories

The Cojeens' Story

Watch to learn more about how the Cojeens navigated Emmett's Duchenne diagnosis and treatment.
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Meet the Marlows

Learn more about Brantley Marlow and how his family chooses to embrace change in the face of his Duchenne diagnosis.
Read Story
The Millers’ Story

Watch to learn about the Miller family and Nathan's life as a teenager with Duchenne
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Ambassador Emmett smiling in front of a navy blue background surrounded by fun iconography.

Helpful Resources

You can download these resources to help guide you and your family through the Duchenne journey.

Insights from Caregivers Who Speak Duchenne

Read these answers to some common questions about living with DMD – all given by caregivers of people with Duchenne.

I thought I was alone, but I realized Duchenne is more common than I thought. I had never heard of it before, but once my son was diagnosed, now we hear about new cases all the time.
-Andrea, mom to Nathan
I learned a few important things. First, not only can Duchenne be inherited from the mother, but it can also happen spontaneously, which was the case with Emmett. Also, the dystrophin gene is the largest human gene, and with 79 exons, so many different deletions can lead to DMD. Plus, when you hear about how dystrophin affects the skeletal muscles, you don't realize how it also affects the heart, lungs and brain.
-Elizabeth, mom to Emmett

As soon as we learned Nathan had Duchenne, we knew we wanted to fight it the best we could. Early on, we connected with an amazing Duchenne advocate and mom and her organization, Team Joseph. She’s passionate, and she inspired us.
-Andrea, mom to Nathan
When Emmett's neurologist first gave me his diagnosis, he brought up exon skipping as a possible treatment because of Emmett’s deletions. We were very hopeful and started to inquire about the treatment right away. We wanted to do anything possible to keep our baby boy healthy and mobile as long as possible.
-Elizabeth, mom to Emmett

Nathan was diagnosed at a great hospital. We decided to visit an official Duchenne center. There we learned about an exon-skipping trial and were very happy to learn that Nathan was one of the boys who could benefit.
-Andrea, mom to Nathan
Our first question after his diagnosis was, are any treatments available? We discussed steroids first, and then his doctor said Emmett qualified for an exon-skipping therapy that would help him produce a shorter version of the dystrophin protein that he needs.
-Elizabeth, mom to Emmett

When we learned about the trial, we didn't care what treatment it was. We wanted to try anything. Nathan was only 7, and we wanted to be aggressive early on. We knew it would be difficult to travel to another city every week, but we felt confident we could make it work; and if Nathan’s body produced even a little dystrophin, the trial would be worth it.
-Andrea, mom to Nathan
When Emmett was first diagnosed in March 2020, we were told only one exon-skipping therapy was available for his deletions, but others were coming in the near future. We wanted to get Emmett on anything that was available right away, so that’s how we started his first exon-skipping therapy. After about a year, our neurologist brought up the idea of switching Emmett to another exon-skipping therapy. She gave us data comparing the two and showing the increased dystrophin production with the other therapy. After looking at the data and reaching out to support groups, we decided to make the switch.
-Elizabeth, mom to Emmett

The therapy was relatively simple. Nathan did whatever the nurses needed him to do, and he never complained about the IV. It took about 2 hours, and he always enjoyed a good lunch! We became very close to his amazing nurses, doctor, and staff. They all took great care of him. The difficult part was the traveling, taking Nathan out of school one day a week and splitting up time with our other son. But we were able to make it work. After the first year and a half, the travel became daunting, so we were thrilled when we were able to start his infusions at home.
-Andrea, mom to Nathan
Besides the roller coaster of emotions waiting for insurance approval, we haven't had any issues starting Emmett on exon-skipping therapy. We have a set routine and aim to do them right around bedtime. We opted to get Emmett a Mediport, which in my opinion was the best decision ever.
-Elizabeth, mom to Emmett

We were fortunate to connect with Team Joseph and other organizations early on, and meet with other Duchenne parents. Social media helped us too, although this became overwhelming, so we’ve pared back on our connections over the years. We became close to a couple of families during the trial, as well, and that truly helped.
-Andrea, mom to Nathan
At first, we didn't know where to look. We did a lot of research online, explored some nonprofit organizations and joined Facebook support groups, and multiple people there reached out to us to share their stories and help us navigate where to go. We’ve also been very involved with PPMD, Parent Project Muscular Dystrophy, and they’ve been a great resource for our family.
-Elizabeth, mom to Emmett