New study tracks early signs of pompe in screened newborns
NCT ID NCT03694561
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 25 times
Summary
This study follows 20 newborns and children who were found to have late-onset Pompe disease through newborn screening. Researchers track muscle and joint symptoms, lab results, and quality of life over several years to learn how the disease develops. No treatments are given; the goal is to better understand the condition and help decide when to start therapies 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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Duke University
Durham, North Carolina, 27705, United States
Conditions
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