One-of-a-Kind genetic treatment trial launches for single child with Ultra-Rare disease
NCT ID NCT07474298
Summary
This study is testing a custom-made genetic medicine for a single child with Schuurs-Hoeijmakers syndrome, a rare disorder that affects development. The goal is to see if the treatment is safe and if it can help improve the child's ability to communicate and use their hands. The treatment is designed to target the specific genetic error that causes this child's condition.
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Contacts and locations
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Locations
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The Hospital for Sick Children (SickKids)
Toronto, Ontario, M5G 1X8, Canada
Conditions
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