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I have 2 children with DMD. One 6 and one 3. We were diagnosed in October of 2024, and we have just found out about a clinical trial that may focus on skipping an exon to put the deletion area back in frame. What are some of the issues to watch for with a clinical trial?
My youngest cant be involved, but my oldest is past the minimum age. We are anxious to hear how others have managed.
Exon-skipping therapy works by helping cells "skip over" missing exons during protein production, essentially patching over gaps to help create functional dystrophin proteins. Before starting any trial, genetic testing is essential to determine which specific exon is missing, as treatments are tailored to target particular Show Full Answer
Hey Bryan,
Our children haven't been offered the Exon-skipping treatment because their missing numbers aren't the approved ones. It has to be that specific number before you're eligible. We tried to get into a gene therapy infusion at Little Rock AR last year but after 2 days of tests, they determined that our oldest, Cayden, wasn't severe enough to get into the trial. I've heard rumors that treatment is hopefully going to be FDA approved and released this fall.
We just opted out of another gene therapy earlier this month after hearing a 16 yr old boy died from liver failure six weeks after the infusion. Scared me and I felt that was a huge red flag for us not to do it.
My advice is talk with your Doctor about your concerns. Do some research yourself about your specific Exon-skipping treatment.
I'd want to know : What side effects have they seen? Have they had any deaths? What about long term side effects? What are the positive results from the clinical trials? How long before seeing any improvement? There's a lot of information online about all of these treatments. Just takes time to research.
We pray every day for a positive treatment or cure for this heart breaking disease. Good luck in your journey! 🙏
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