Gene therapy breakthrough for rare childhood disease?
NCT ID NCT06149403
First seen Jan 07, 2026 · Last updated May 25, 2026 · Updated 25 times
Summary
This study tests a new gene therapy, OTL-203, for children with Hurler syndrome, a rare genetic disease that damages organs and the brain. It compares the gene therapy to the current standard treatment, a stem cell transplant. The goal is to see if the gene therapy can prevent death, the need for another transplant, or severe health problems.
Disclaimer
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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.
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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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
Explore the condition pages connected to this study.