Gene therapy offers hope for rare blood disorder
NCT ID NCT06422351
First seen Apr 25, 2026 · Last updated May 05, 2026 · Updated 3 times
Summary
This study tests a gene therapy for people with pyruvate kinase deficiency, a rare blood disorder causing severe anemia. About 10 participants will receive an infusion of their own blood stem cells that have been modified to produce a working version of the missing enzyme. The goal is to raise hemoglobin levels and reduce the need for blood transfusions.
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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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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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