One-of-a-Kind drug created for single Patient's devastating genetic illness
NCT ID NCT07221760
Summary
This study is creating a custom drug for one person with a rare, inherited brain disease called DRPLA. The goal is to see if this personalized treatment is safe and can help control the patient's seizures and other symptoms. Researchers will closely monitor the patient's health and quality of life for up to two years after treatment.
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Contacts and locations
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Locations
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Dell Children's
Austin, Texas, 78723, United States
Conditions
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