One-of-a-Kind treatment created for single Child's unique genetic disease
NCT ID NCT07197294
Summary
This study is creating and testing a personalized drug for a single child with a rare genetic disorder called NEDBA. The drug is designed to target the child's specific genetic mutation. Researchers will check if the treatment is safe and if it helps improve the child's movement, seizures, and overall quality of life over two years.
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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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