AxD Outcomes
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Description
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.
Eligibility and criteria
IRB Number:
16-012649
Clinical trial phase:
N/A
Official title:
Related specialties
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