Tracking a hopeful gene therapy for a devastating childhood disease
NCT ID NCT04273243
Summary
This study is tracking 10 children with CLN6 Batten disease for up to 5 years after they received a single, experimental gene therapy. The goal is to understand the long-term safety and whether the treatment continues to help slow the progression of this fatal neurological disorder. Researchers will monitor the children's health and measure changes in their motor and language skills over time.
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Contacts and locations
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Locations
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Nationwide Children's Hosptial
Columbus, Ohio, 43205, United States
Conditions
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