Early step toward a genetic fix for sickle cell
NCT ID NCT02212535
Summary
This small, early-stage study tested the safety and effectiveness of a single injection of a drug called plerixafor. The goal was to see if it could safely move stem cells from the bone marrow into the bloodstream of adults with severe sickle cell disease, so those cells could be collected. These collected cells could then potentially be used in a future gene therapy treatment, which would aim to control the disease.
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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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Hôpital Necker - Enfants Malades
Paris, 75015, France
Conditions
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