One-of-a-Kind drug created for single child in groundbreaking trial
NCT ID NCT07197294
Summary
This study is testing a custom-made drug designed for a single child with a rare genetic disorder called NEDBA, which affects brain development and movement. The goal is to see if the treatment is safe and if it can help improve the child's motor skills, reduce seizures, and enhance quality of life over two years. This is an early-phase trial focused on one participant to gather initial data.
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Contacts and locations
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Locations
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Columbia University
New York, New York, 10032, United States
Conditions
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