One-of-a-Kind drug created for single Child's devastating genetic illness
NCT ID NCT07222371
Summary
This study is testing a custom-made genetic drug for a single child with a rare, severe brain disorder called TUBB4A-related leukodystrophy. The drug is designed to target the child's specific genetic mutation, with the goal of controlling symptoms like muscle stiffness and improving motor function. Researchers will carefully monitor the child's movement, abilities, and quality of life to see if the treatment helps.
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Contacts and locations
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Locations
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Rady Children's Hospital San Diego
San Diego, California, 92123, United States
Conditions
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