Gene therapy for rare blood disease shows promise in Long-Term Follow-Up
NCT ID NCT04437771
First seen Apr 14, 2026 · Last updated May 16, 2026 · Updated 5 times
Summary
This study checks on the long-term safety and effectiveness of a gene therapy for people with Fanconi Anemia, a rare genetic disorder that affects the bone marrow. Nine patients who received the therapy in an earlier study will be followed for 15 years to see if their blood counts stay stable and if there are any side effects. No new treatment is given during this follow-up period.
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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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Hospital Infantil Universitario Niño Jesús (HIUNJ)
Madrid, 28009, Spain
Conditions
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