STAT News: In Duchenne Muscular Dystrophy, a Promising New Therapy Is Available to a Fortunate Few
"The mutations Dyne and Novartis have said they’re pursuing could help just 35% to 40% of Duchenne patients. Many other mutations that could be treated with this technology are unique to just a few patients. These parents assume companies will never have interest in developing drugs for their children, at least not in time."
This is a story about a new drug for Duchenne on the verge of approval—it builds upon the science behind the first-ever drug for the disease. For some boys, it will turn their Duchenne into a manageable chronic condition. It will give them lab values in normal ranges they’ve never had. For some, like Brecken, it will enable them to walk for miles; for others, like Yannick, it could dramatically slow their daily decline. It will fill minds with new possibilities for the future.
Charlie is not one of those children. He’s not Brecken in this story, or even Yannick, who is waiting for his mutation to come up next. Charlie is a boy born with the terrible genetic luck of a rare disease with a mutation these companies will never touch. To them, he's a small n=. To us, an entire world. A minds-eye montage of birthdays, holidays, everydays that could be if we could just align the stars.
So align them we will. As long as we have legs we will walk them into every room that contains the power to change bad odds with good science. As long as we have working minds we will find the right ones to solve this disparity of DNA. As long as we have voices we will advocate that every life is worthy of human-powered solutions when science does its astonishing, humbling thing.
Charlie knows Brecken. He loves the way he says “okay” in his sweet Minnesota accent. They met in Cincinnati, the fun there ancillary to a visit with a Duchenne cardiologist. Brecken showed Charlie his scooter, and in an instant Charlie felt more comfortable with the idea of getting one to use for long distances, too. What if the next time they meet they can compare golf swings instead of assistive devices?
This drug can do that. And so we will push back on a system that says certain boys’ future isn’t worth the trouble or cost.
I am endlessly grateful to Brecken's mom, my dear friend, for sharing this story and fighting for Charlie, and Yannick and all boys who might benefit from this meaningful—yet currently limited—progress, just as she has for her own. Stay tuned. We have plans for Charlie and they don’t include giving up just because it’s hard.
Read more at STAT NEWS: https://www.statnews.com/2026/10/07/muscular-dystrophy-exon-skipping-drugs-novartis-dyne/