One-Shot gene therapy trial offers hope for devastating childhood disorder
NCT ID NCT07480564
Summary
This study is testing a single injection of an experimental gene therapy called TSHA-102 in three young girls (aged 2 to under 4 years) with Rett syndrome. The goal is to see if this one-time treatment is safe and if it can help control the symptoms of this serious genetic disorder. Researchers will follow the girls for five years after the injection to monitor their health and development.
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Genom att skicka in godkänner du våra Användarvillkor
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Genom att skicka in godkänner du våra Användarvillkor
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