New stem cell approach aims to tame rare genetic diseases
NCT ID NCT02171104
First seen Jan 09, 2026 · Last updated May 11, 2026 · Updated 12 times
Summary
This study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get donor cells to grow in the patient's body while keeping transplant-related deaths low. Participants receive chemotherapy drugs (busulfan and fludarabine) before the transplant, with drug levels carefully monitored. The study includes children and adults up to age 55.
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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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Locations
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Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455, United States
Conditions
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