Gene therapy survivors monitored for decades in landmark study
NCT ID NCT04049084
First seen Dec 18, 2025 · Last updated May 15, 2026 · Updated 27 times
Summary
This study follows about 70 people who previously received gene therapy for a rare immune disorder called ADA-SCID. Researchers want to see how well the treatment worked over time and check for any long-term side effects. No new treatment is given in this study—it only collects information from medical records and checkups.
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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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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
Explore the condition pages connected to this study.