Custom gene-targeting drugs offer hope for kids with ultra-rare brain diseases
NCT ID NCT07410143
Summary
This study tests a custom-made genetic medicine called an antisense oligonucleotide (ASO) for one child with a rare, life-threatening brain disorder caused by a unique genetic mistake. The ASO is designed to fix that specific error. The main goal is to check if the treatment is safe and tolerable, while also measuring how it 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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