A thorough review of outcome measures that assess disease progression has not been established for any of the subcategories of Alexander disease. These results will be crucial to our understanding of disease presentation and progression. Providing this information about the pathophysiology will allow for effective development of clinical trials to test therapies and the future use of neuroprotective agents in children.

We want to study brain responses during simulated driving and are currently looking for participants 12-17 years old or those who have had brain surgery. To do so, we'll have eligible participants come to CHOP for some non-invasive (safe) brain scans and drive using a simulator. If you are interested in learning more about the study, please contact Victoria Kaufman at kaufmanv@chop.edu or 267-425-4150.