Evaluation of Outcome Metrics in Alexander Disease
Participants will be asked to complete physical examinations including physical therapy, occupational therapy, speech and language therapy, and swallowing assessments. Patients (or caretakers) may be asked to complete questionnaires as well. The study asks for participants to return at least once yearly to repeat assessments.
Purpose of Study
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.
Key Inclusion / Exclusion Criteria
- Diagnosed with Alexander Disease
- Other Leukodystrophies will not be enrolled
Clinical Trials ID
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Start Date / End Date:
01 2016 - 12 2026