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Krabbe Disease Global Patient Registry
Sponsor: State University of New York at Buffalo
Summary
The purpose of this study is to develop a clinical database of individuals diagnosed with Krabbe disease in order to determine which symptoms herald the onset of clinical disease in the various phenotypes of Krabbe disease; to determine whether level of GALC enzyme activity, or a specific genetic mutation predict the clinical course; and to determine which neurodiagnostic tests predict onset and/or severity of the disease.
Official title: The Institute for Myelin and Glia Exploration's Clinical Database of Patients With Krabbe Disease, A World-Wide Registry
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
60
Start Date
2014-09
Completion Date
2026-09
Last Updated
2025-02-10
Healthy Volunteers
No
Conditions
Locations (1)
State University of New York at Buffalo
Buffalo, New York, United States