New hope for kids with devastating genetic diseases
NCT ID NCT02171104
Summary
This study is testing a stem cell transplant procedure for children and adults with severe, inherited metabolic disorders and a rare bone disease. The goal is to see if using a specific drug combination can help the donor cells successfully take hold in the patient's body while keeping the risks of the transplant low. Researchers are measuring how well the transplant works and monitoring the patient's disease progression afterward.
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Contacts and locations
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Locations
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Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455, United States
Conditions
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