Harrison's story: How gene therapy could help against Duchenne muscular dystrophy

When Pam first heard about gene therapy, she thought it sounded like science fiction. She never would have guessed her son would be among the first patients to receive an innovative new gene therapy that's giving new hope against Duchenne muscular dystrophy (DMD).

Their journey started with a phone call that would change their family forever.

Welcoming Harrison into the family

In addition to having six biological children, Pam and her husband, Bobby, are foster parents. It was their case manager who called that night in 2019. She had a 6-month-old child who needed not just a foster home, but an adoptive home. This child had Duchenne muscular dystrophy.

DMD happens because of a genetic change that causes a child to be unable to make a protein called dystrophin. Without this protein, muscles break down and become weak all over the body. This limits a child's mobility more and more as they grow up and, ultimately, affects the heart muscle and muscles important for breathing. People with this diagnosis rarely live to 30.

Pam and Bobby stayed up all night thinking about this baby who needed a family. The next day, they talked to their children.

"We knew that this would be a huge undertaking, but we all felt that we couldn't know about this child and say no," Pam says. "Everybody was on board. It was scary but also exciting. And now he is just so loved by our older kids. It's precious to watch all of them together."

Learning to care for a child with complex medical needs

Harrison was soon getting acquainted with his new family, while Pam and Bobby learned what his everyday care looked like. Pam got to know a wide range of doctors at the Duchenne Muscular Dystrophy (DMD) Program at Children's Health℠, where Harrison received care from neurologists, physical therapists, cardiologists, pulmonologists and other experts.

"Our story is different from a lot of other families because there wasn't this moment where we got a devastating diagnosis," Pam says. "We knew about it from the beginning and knew that he would miss many milestones. So instead of feeling sad about the milestones he missed, we celebrated every milestone he met."

Harrison never crawled and learned to walk a bit later than most kids, around age 2. At age 4, he still has trouble running and jumping, but he loves swimming and splashing in the pool.

Still, their family is well aware of the outlook for kids with DMD. Since the disease causes significant muscle degeneration, many kids are reliant on a wheelchair by age 12. Without treatment, they will need help brushing their teeth and eating because of limited mobility in their arms. DMD also makes it difficult for their heart and lungs to work. Current treatments using corticosteroids can slow the disease but won't stop or cure it.

Last year, their doctor told them about the gene therapy and how it slows down DMD by targeting the root cause of the disease. It brought Pam full circle to when she first heard about gene therapy from her daughter, who learned about it in biology class. What seemed so futuristic was now real and had the potential to help Harrison. Their family was all in.

A potentially game-changing gene therapy

This new medicine aims to help the body make a better version of the protein that doesn't work properly, so children can maintain the muscles they need to grow and thrive. It's currently given just one time through an IV for boys aged 4 and up.

"This treatment is still very new, but anecdotally we've seen kids climbing and running around and getting stronger instead of weaker, which is almost unheard of in DMD. Parents are seeing their kids experiencing something closer to the childhood they expected before the diagnosis," says Kaitlin Batley, M.D., Pediatric Neurologist and Director of the Duchenne Muscular Dystrophy Program at Children's Health.

In October 2023, Harrison was the first Children's Health patient to receive this treatment and Children's Health is one of the few hospitals currently offering it. Harrison is one of the many kids that Children's Health has helped through giving them early access to game-changing new therapies soon after approval.

"I can't put into words how fortunate we feel to be a part of this," Pam says. "I feel like we're standing on the shoulders of the families who have been dealing with this for so many years, and we were in just the right place at the right time to have this treatment available to Harrison."

A safe place and a hopeful future

While Pam and Bobby are veteran parents, Harrison is their first child with complex medical needs. They feel lucky to have Children's Health on their side.

"I remember first walking in these doors and just feeling the weight of being at a children's hospital," Pam says. "People are here because they're hurting for a lot of reasons, but they came to find healing. Every time we meet with a doctor we realize just how fortunate we are to be in a place where such amazing work is happening. It can be really heavy to be a parent of a medically fragile child or a special needs kiddo, so we're grateful to have a safe place in Children's."

Since receiving the treatment, Harrison has gotten better and better. He's walking easier, running, climbing and even jumping a little.

"Families like Harrison's who push the boundaries and receive these innovative medications early on make big advances possible," Dr. Batley says. "I'm excited about what the future holds and I hope that this work will open the door for DMD to one day be a completely different and much less devastating diagnosis. Harrison is such a positive, playful kid. He's this ray of sunshine and he doesn't let anything slow him down. He won't quit and neither will we."

Learn more

A recognized leader in treating muscular dystrophy, Children's Health uses the latest research to extend and improve the lives of children with muscular dystrophy. Our team of neuromuscular, respiratory, heart and physical therapy experts work together to provide the best care and help children stay strong as they grow up. Learn more about our comprehensive muscular dystrophy care.