Tiny study tracks enzyme levels in kids with rare disease before and after transplant
NCT ID NCT05634512
First seen Nov 01, 2025 · Last updated Apr 30, 2026 · Updated 23 times
Summary
This study looks at how children with a rare genetic disease called MPS IH process the enzyme drug laronidase before and after a stem cell transplant. Researchers will collect blood samples from 13 children up to 3 years old to see how factors like body size affect drug levels. The goal is to learn more about how the transplant changes the way the drug works, which may help doctors give the right dose in the future.
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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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University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455, United States
Conditions
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