Study seeks to measure how treatment affects development in devastating childhood disease
NCT ID NCT03862274
Summary
This study aims to understand how a rare, fatal childhood brain disease called CLN2 Batten disease affects children's development. Researchers will track 30 children, comparing those receiving a treatment called cerliponase alfa to those who are untreated. The goal is to measure changes in skills like language, movement, and problem-solving to better understand the disease and the treatment's effects.
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Contacts and locations
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Conditions
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