First-Ever gene therapy trial launches for devastating childhood nerve disease

This is the first human study to test a single-dose gene therapy called ELP-02 for children and young adults (ages 3-20) with a rare, inherited nerve disease called Charcot-Marie-Tooth type 4J (CMT4J). The therapy aims to deliver a working copy of a faulty gene directly to the nervous system to slow or stop disease progression. The main goals are to check if the treatment is safe and to see if it can improve or stabilize muscle function, mobility, and quality of life.