Experimental gene therapy aims to fix rare childhood disease
NCT ID NCT03488394
First seen Jan 11, 2026 · Last updated May 13, 2026 · Updated 17 times
Summary
This study tests a new gene therapy for children with MPS I Hurler, a rare genetic disease that damages organs. The treatment takes the child's own blood stem cells, adds a working copy of the missing gene, and puts them back. The goal is to help the body produce the missing enzyme and control the disease long-term.
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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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Locations
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Ospedale San Raffaele
Milan, 20132, Italy
Conditions
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