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A Natural History Study of the Gangliosidoses
Sponsor: University of Minnesota
Summary
Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases. This research study seeks to develop a quantitative method to delineate disease progression for the gangliosidosis diseases (Tay-Sachs disease, Sandhoff disease, and GM1 gangliosidosis) in order to better understand the natural history and heterogeneity of these diseases. Such a quantitative method will also be essential for evaluating any treatments that may become available in the future, such as gene therapy. The data from this study will be necessary to provide end-points for future therapies, guide medical decisions about treatment, provide objective measurement of treatment outcomes, and accurately inform parents regarding potential outcomes.
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
52
Start Date
2010-12
Completion Date
2027-03-01
Last Updated
2026-03-04
Healthy Volunteers
No
Conditions
Locations (1)
University of Minnesota - Pediatric Genetics and Metabolism
Minneapolis, Minnesota, United States