Gene therapy offers hope for children with rare brain disease

This study tested a gene therapy for children with metachromatic leukodystrophy (MLD), a rare, inherited brain disease that causes severe disability and early death. The treatment uses the child's own blood stem cells, which are modified in a lab to carry a working copy of the missing gene, then given back to the child. The goal was to slow or stop the disease from getting worse. Twenty children took part, and the results showed that the therapy could improve motor function and survival compared to untreated patients.