Gene therapy trial offers hope for cure of devastating childhood disease
NCT ID NCT06149403
Summary
This study is testing whether a new gene therapy called OTL-203 works better and is safer than the current standard treatment (stem cell transplant) for children with Hurler syndrome. Hurler syndrome is a severe genetic disorder that causes progressive damage to the brain, bones, and organs. The trial will follow 40 children for up to 5 years to see which treatment helps them live longer without major complications.
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Contacts and locations
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Locations
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Manchester University NHS Foundation Trust Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
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Ospedale San Raffaele
Milan, 20131, Italy
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Princess Maxima Center
Utrecht, 3584 CS, Netherlands
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UMC Utrecht
Utrecht, 3584 CX, Netherlands
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University of Minnesota, Pediatrics
Minneapolis, Minnesota, 55455, United States
Conditions
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