Gene therapy for rare immune disease shows promise in Long-Term Follow-Up
NCT ID NCT06282432
First seen Jan 06, 2026 · Last updated May 22, 2026 · Updated 21 times
Summary
This study follows 9 people with a rare immune disorder called LAD-I who previously received a one-time gene therapy. The goal is to see if the treatment remains safe and effective over time, and whether it helps them avoid needing a stem cell transplant. Researchers will track infections, hospital stays, and skin rashes to measure success.
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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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Contacts and locations
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Locations
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Hospital Infantil Universitario Niño Jesús
Madrid, 28009, Spain
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University College London Great Ormond Street Institute of Child Health (GOSH)
London, WC1N 1EH, United Kingdom
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095-1489, United States
Conditions
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