Gene therapy breakthrough offers hope for boys with rare brain disease
NCT ID NCT05665166
First seen Apr 02, 2026 · Last updated May 24, 2026 · Updated 9 times
Summary
This study tests a one-time gene therapy for boys with Hunter syndrome, a genetic disorder that damages the brain and body. The therapy uses the child's own blood stem cells to produce the missing enzyme, aiming to stop disease progression. Five boys aged 3 to 22 months will be enrolled and followed for 2 years to check safety and effectiveness.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Manchester University Foundation Trust
Manchester, United Kingdom
Conditions
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