Tiny study tracks enzyme levels in kids with rare disease before and after transplant
NCT ID NCT05634512
First seen Nov 01, 2025 · Last updated Jun 18, 2026 · Updated 32 times
Summary
This study watches how the enzyme drug laronidase moves through the bodies of 13 children with MPS IH (a rare genetic disease) before and after they get a stem cell transplant. The goal is to learn what affects drug levels, not to test a new treatment. Children aged 0-3 who are already scheduled for transplant and enzyme therapy can join.
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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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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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