Custom Gene-Targeting drug tested for rare developmental disorder
NCT ID NCT07474298
First seen Mar 18, 2026 · Last updated May 22, 2026 · Updated 8 times
Summary
This study tests a custom-made drug designed for one person with Schuurs-Hoeijmakers syndrome, a rare genetic condition that affects development. The drug, called an antisense oligonucleotide, aims to correct the effects of a specific gene mutation. The trial will measure changes in communication and motor skills over two years.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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