Clinical Trial: Natural History and Outcome Measures in Alexander Disease

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational




Official Title: Natural History and Outcome Measures in Alexander Disease

Brief Summary: The purpose of this study is to define the natural history of Alexander Disease, a leukodystrophy that causes neurological dysfunction. Investigators will obtain clinical outcome assessments to measure how the disease affects a patient's gross motor, fine motor, speech and language function, swallowing, and quality of life. The data obtained from this study will be used for the design of future treatment trials.