Doctors craft One-of-a-Kind gene therapy for Child's devastating brain disease
NCT ID NCT06369974
Summary
This study is testing a custom-made genetic medicine for a single child with a severe, rare brain disease called TUBB4A leukodystrophy. The goal is to see if the personalized treatment is safe and can help control the disease by improving the child's movement, development, and quality of life. Researchers will carefully monitor the child's progress over two years after receiving the experimental drug.
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Contacts and locations
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Locations
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Conditions
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