Gene therapy for rare blood disease tracked for 15 years
NCT ID NCT04437771
First seen Apr 14, 2026 · Last updated May 22, 2026 · Updated 7 times
Summary
This study follows 9 people with Fanconi Anemia who received a one-time gene therapy to fix a faulty gene. Researchers will monitor their health and blood counts for 15 years to see if the treatment remains safe and effective. No new treatment is given during this follow-up period.
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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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Hospital Infantil Universitario Niño Jesús (HIUNJ)
Madrid, 28009, Spain
Conditions
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