Researchers launch major study to track rare brain disease over time

This study follows up to 600 people with leukodystrophy, a rare genetic disorder affecting the brain's white matter. Researchers aim to track how the disease progresses, improve diagnosis through genetic testing and brain scans, and evaluate outcomes like hospitalizations and response to treatments such as bone marrow transplant. No new drug or therapy is being tested; instead, the goal is to gather knowledge to better care for patients.