Alexander Disease (AxD) Research
This study gathers natural history information about your child’s Alexander disease (AxD) progression through a telephone questionnaire. This study is for those families unable to travel to Children’s Hospital of Philadelphia (CHOP). No visit to CHOP is required.
Participants who are able to travel to CHOP may be invited to participate in additional evaluations to explore Alexander disease outcomes through a series of on-site physical therapy, occupational therapy, speech and swallowing evaluations.
If you or your child has a confirmed diagnosis of Alexander disease and you are interested in learning more about this study, please contact study coordinator Geraldine Liu at LiuG@email.chop.edu.
More leukodystrophy natural history studies
Our experts in the Leukodystrophy Center of Excellence are currently working on a number of natural history studies for specific leukodystrophies. A natural history study is a carefully designed research study that follows a group of people over time who have a specific medical condition or disease. Researchers working on these studies collect specific health information and biological samples in order to understand how the disease develops and how to treat it. See a full list of actively enrolling studies.