Alexander disease, a Leukodystrophy, is a progressive and fatal neurodegenerative disease. It is a rare genetic disorder and mostly affects infants and children, causing developmental delay and changes in physical characteristics.
Current research is aimed at understanding the mechanisms by which the mutations cause disease and exploring potential strategies for treatment.
Clinical Trial - Natural History Intake
Children’s Hospital of Philadelphia (CHOP) is actively seeking Alexander Disease patients for the Natural History intake clinical trial. The data collected over the next 10 years will provide a picture of what the disease progression looks like prior to any treatment.