AxD Outcomes

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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.

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at liug [at] chop.edu or 267-425-2063.

Eligibility & Criteria

IRB #:
16-012649
Official Title:
Evaluation of Outcome Metrics in Alexander Disease
Study Phase:
Not Applicable
Eligible Age Range:
Any
Gender:
All
Study Categories:
Leader
Amy T. Waldman

Amy Waldman, MD, MSCE