Pompe disease patients tracked to unlock immune therapy secrets
NCT ID NCT01665326
First seen Apr 14, 2026 · Last updated Jun 16, 2026 · Updated 13 times
Summary
This study follows up to 400 people with Pompe disease over many years to see how their immune system reacts to standard enzyme replacement therapy. Researchers collect medical records and lab results to learn why some patients develop antibodies that reduce treatment effectiveness. The goal is to better understand the disease and improve future care, but no new treatment is being tested.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Duke University Medical Center
RECRUITINGDurham, North Carolina, 27710, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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