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A new clinical trial is offering hope for people with Charcot-Marie-Tooth (CMT) disease. Researchers tested a plasmid-based gene therapy designed to support nerve and muscle function.

Unlike viral gene therapies, this treatment can be given multiple times without triggering an immune response—making it a promising option for chronic conditions like CMT.

In a small Phase 1 trial in Seoul, South Korea, 12 participants with CMT type 1A received injections of the therapy over 104 days. The results? No serious side effects—just mild, temporary itching and swelling. Importantly, no one developed antibodies against the treatment, meaning their bodies accepted it without issue.

And the best part—participants showed signs of improvement. Their CMT Neuropathy Score improved by an average of 2.17 points and seven out of 12 showed better functional ability. Additionally, four participants improved in leg movement, and walking speed slightly increased.

These early results are promising, and researchers are already planning a larger Phase 2 study to see if the treatment can help more people. While there’s still a long road ahead, this trial offers real hope for a future where living with CMT gets easier.