First-of-its-Kind trial offers bespoke genetic medicine for child with devastating brain disease
NCT ID NCT07410143
Summary
This early-phase trial is testing a new, personalized type of medicine for children with rare, severe, and life-threatening brain conditions caused by unique genetic errors. The medicine, called an antisense oligonucleotide (ASO), is custom-designed for a single participant's specific genetic problem. The main goals are to check if the treatment is safe and to see how the drug moves through the body.
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Contacts and locations
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Locations
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Great Ormond Street Hospital
London, United Kingdom
Conditions
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