Gene therapy could free patients from frequent blood transfusions
NCT ID NCT06422351
First seen Apr 25, 2026 · Last updated May 24, 2026 · Updated 5 times
Summary
This trial tests a one-time gene therapy for people with pyruvate kinase deficiency, a rare blood disorder causing severe anemia. Patients receive their own blood stem cells modified to produce a working enzyme. The goal is to raise hemoglobin levels and reduce or eliminate the need for regular blood transfusions. The study is currently on hold.
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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
Madrid, 28009, Spain
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Hospital Universitario Fundación Jiménez Díaz
Madrid, Spain
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Stanford University
Palo Alto, California, 94305, United States
Conditions
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