New Gene-Based injection tested for nerve disease safety

This early-stage trial tested whether an experimental drug called Engensis (VM202) is safe when injected into the leg muscles of people with Charcot-Marie-Tooth disease type 1A (CMT1A), a genetic nerve disorder that causes muscle weakness. Twelve adults with mild to moderate CMT1A received multiple injections over 270 days. The main goal was to check for side effects, while researchers also looked for any signs of improvement in nerve function.