Gene therapy survivors monitored for decades in landmark safety study
NCT ID NCT04049084
First seen Dec 18, 2025 · Last updated May 19, 2026 · Updated 28 times
Summary
This study follows 70 people who previously received gene therapy for a rare immune disorder called ADA-SCID. Researchers will track their health, survival, and any side effects over many years. No new treatment is given—this is purely an observation to see how well the gene therapy holds up long-term.
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the original study
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center
Los Angeles, California, 90095, United States
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UCL Great Ormond Street Institute of Child Health
London, WC1N 1EH, United Kingdom
Conditions
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