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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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.
Contacts and locations
Locations
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Manchester University Foundation Trust
Manchester, United Kingdom
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Other studies related to the condition(s) this trial covers.
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