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Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae
Sponsor: Emily de los Reyes
Summary
CLN6 is a rare, neurodegenerative disease that causes progressive loss of acquired skills with motor delay, visual loss, seizures and ataxia. The investigators propose a natural history study of this rare disorder since it is currently unknown. It is important to understand disease progression in CLN6 disease to be able to judge therapeutic efficacy as emerging therapies like gene therapy become available.
Official title: Natural History Study of Batten's CLN6 Disease
Key Details
Gender
All
Age Range
2 Years - 25 Years
Study Type
OBSERVATIONAL
Enrollment
30
Start Date
2017-01
Completion Date
2026-12
Last Updated
2025-07-30
Healthy Volunteers
No
Conditions
Interventions
Natural history
Parent interview
Locations (1)
Nationwide Children's Hospital
Columbus, Ohio, United States