One-Shot gene therapy aims to cure devastating childhood disease
NCT ID NCT06149403
Summary
This study is testing if a single gene therapy treatment (OTL-203) can provide a lasting cure for children with Hurler syndrome, a severe genetic disorder. It directly compares this new approach to the current standard treatment, which is a stem cell transplant from a donor. Researchers will follow 40 children for up to 5 years to see which treatment is safer and more effective at keeping them alive and healthy.
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