One-Time gene therapy aims to halt devastating childhood brain disease

This study is testing a single infusion of an experimental gene therapy called OTL-200 for children and teenagers with a rare, progressive brain disease called Late Juvenile Metachromatic Leukodystrophy (MLD). The therapy involves collecting a patient's own blood stem cells, adding a working copy of a missing gene in a lab, and then returning the modified cells to the patient. The main goal is to see if this one-time treatment can safely slow or stop the progression of the disease by restoring a crucial missing enzyme in the brain and nervous system.