Experimental stem cell infusion tested for rare nerve disorder

This early study tested a single intravenous dose of umbilical cord-derived stem cells (EN001) in 3 adults with Charcot-Marie-Tooth disease type 1E, a severe hereditary nerve condition that causes muscle wasting, breathing problems, and often wheelchair dependence. The main goal was to check safety, with a secondary look at whether the treatment might slow disease progression. Previous studies in a related CMT type found no serious side effects, but this small trial cannot prove effectiveness.