Ophélie Vacca, Cathy Nagy, Aurélie Goyenvalle. Beyond Muscle: Delivering RNA therapeutics to the CNS in Duchenne Muscular Dystrophy. Journal of neuromuscular diseases. 2025

https://doi.org/10.1177/22143602251360668 

Duchenne muscular dystrophy (DMD) is a genetic disease that causes muscles to weaken over time, leading to difficulties with walking, breathing, and heart function. But DMD doesn’t just affect muscles; the brain can also be involved, leading to problems with learning, memory, and behaviour. Current treatments focus mainly on the muscles, and do not fully address these brain-related challenges.

This article reviews new research on using RNA-based medicines, which can “fix” the faulty dystrophin gene, not only in muscle but also in the brain. The big challenge is that it’s hard to get these medicines into the brain because of protective barriers. Scientists are testing new ways to deliver them, like directly into the spinal fluid or by designing therapies that can cross into the brain more easily. If successful, these approaches could improve both the physical and cognitive symptoms of DMD, leading to better overall care for patients.