MIDDLETOWN, Ohio — While Tristan and Anderson Hayes may have a few years between them, they get along like most brothers do: playing video games, sharing laughs and occasionally getting on each other's nerves.
But the Middletown duo have a rare bond shared by few others in the entire world.
"Some days it's hard," their mother Rachel said. "It was like a punch to the chest, basically. You're not expecting it, but we knew with Anderson, when I was pregnant with him, there was a 50/50 chance of him getting it."
Tristan, 19, and Anderson, 11, have Duchenne muscular dystrophy. It's a rare and deadly degenerative disease that can affect every muscle in the body, including the heart and lungs.
Duchenne is the most common and severe form of muscular dystrophy, caused by loss of dystrophin beneath the sarcolemma, which is the transparent sheath surrounding the fibers of skeletal muscles.
The brothers are two of 15,000 boys living with the disease in the United States and among 300,000 globally. Most boys who have it do not survive beyond their late twenties, and those who do use a wheelchair by age 12.
Though mostly impacting boys, the disease can also be diagnosed in girls, Rachel said.
"Though it's rare," she said. "And it's not just genetic. You can have a spontaneous mutation. If it's never been in your family before your child can still get diagnosed with it."
Tristan was diagnosed around 4 years old and Anderson was diagnosed shortly after birth. Their DMD has progressed since then and both are now in wheelchairs.
"It was heartbreaking," said their father Doug. "You don't want to see your kids have to go through something like that."
Living longer with DMD, Tristan's outlook is less optimistic than his younger brother's. In fact, he doesn't like to think about the disease at all, he said.
"It's not really fun once you think about it ... but I'm working with it as much as I can," he said. "(The biggest challenge) is not thinking about it."
There's an added challenge to the brothers' cases. Not only is there no cure for Duchenne, but Tristan and Anderson have a unique mutation that limits their access to current clinical trials and treatments.
"It's one of the rare forms of Duchenne," Rachel said. "It's harder to find drugs for them because of the rarity of it. But you can't lose hope. It wouldn't put you down a good path if you lost hope."
The Hayes continue to hold on to hope, a mission that's emboldened by their network of fellow Duchenne families.
There's a handful in the Tri-State, they said, including in Monroe, Cincinnati and Northern Kentucky.
"It's nice to have people that close. We've gotten together with a few of them," Doug said. "You feel a little more normal. They're going through the same thing we are."
But the Hayes are also members of an even larger Duchenne Family. They've been connected with the global nonprofit CureDuchenne for years now.
The organization focuses on improving and extending the lives of people with DMD, as well as funding innovative research to find treatments. CureDuchenne created the first Duchenne-specific physical therapy program and funds studies for human clinical trials for eteplirsen, the first-ever FDA-approved drug for Duchenne patients.
"When my family was diagnosed in 2017, there was somebody already working on this," said Leslie Porter, community engagement coordinator with CureDuchenne.
When the organization was founded 20 years ago, there was no research nor any treatment for Duchenne, Porter said. Now, there are new gene-based therapies with noted advances in gene replacement strategies, RNA-based technology, and pharmacological approaches.
Porter's own son was diagnosed with DMD at 6 years old.
"To think that there is this devastating disease that is just going to take away all the beauty of our kids, it's a devastating diagnosis to receive," she said. "We were told, 'Go home, love your kid, come back when you need a wheelchair.' So we left and went home with no hope."
Porter said that hope returned with CureDuchenne.
The organization hosts events and webinars throughout the year, inviting Duchenne families and other community members to learn about updates on research and clinical trials, family support, physical therapy and medical equipment and current standards of care.
"When you think about muscle, when you think about how much of our bodies that impacts — it is overwhelming, it's a lot to take on," Porter said. "But I'm so grateful and it fills me with such hope to know science is actually getting steam behind this and not giving up on this."
The next workshop will be held in Cincinnati on Saturday featuring top experts from CureDuchenne as well as Cincinnati Children’s Hospital and the University of Cincinnati.
The free, full-day workshop runs from 8 a.m. to 4 p.m. at the Westin in Cincinnati. You can register here.
CureDuchenne partners with Children's as the hospital's neuromuscular cardiomyopathy program is one of the leading clinics treating muscular dystrophy in the country.
"I mean people come from all over the country for Cincinnati so we're lucky to have it that close," Doug said.
The Hayes said they're looking forward to this weekend's workshop, specifically to get more insight on current drugs in the pipeline and more standardized care options
"We just want to find a cure in their lifetime," Doug said. "And I hope with the research and the trials going on that we can make that happen."
"I know that not everyone is impacted by this disease but the research and the science that is being discovered now is going to help and impact and change all kinds of medical fields, it's not just going to be used in this disease," Porter said.
Until a cure is found, the Hayes said they will continue on like any normal family.
For Tristan, that means playing video games, drawing, watching anime and sometimes taking a stroll around the block.
For Anderson, the family jokster, that means making his family laugh, poking fun at Tristan, and playing video games while gearing up to be a YouTuber.
Both will continue to dream big, hoping one day a cure will finally become reality.
Then the sky's the limit, even if that "sky" is a simple action we all take for granted.
"I could use an escalator," Tristan said. "(That's something I really want to do.)"
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