Gene therapy breakthrough offers hope for boys with rare brain disease
NCT ID NCT05665166
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new 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, modified to produce the missing enzyme, and aims to stop disease progression. Five boys aged 3 to 22 months will be treated 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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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Locations
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Manchester University Foundation Trust
Manchester, United Kingdom