Scientists track rare disease drug in tiniest patients
NCT ID NCT05634512
Summary
This study aims to understand how a drug called laronidase works in very young children with a rare genetic disorder (MPS I) who are getting a stem cell transplant. Researchers will observe and measure the drug levels in the blood before and after the transplant. The children will receive the drug and transplant as part of their standard medical care; the study just adds extra blood draws to learn more.
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Contacts and locations
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Locations
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University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455, United States
Conditions
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