Gene therapy after stem cell transplant shows promise for rare brain disease

This study tests a one-time gene therapy infusion (FBX-101) given after a standard stem cell transplant in 6 children with infantile Krabbe disease, a severe genetic disorder affecting the nervous system. The therapy uses a harmless virus to deliver a working copy of the GALC gene, aiming to improve motor skills like sitting independently. The main goals are safety and checking whether the treatment helps children achieve better movement compared to those who only had a stem cell transplant.