Pompe disease study tracks immune reactions to enzyme therapy
NCT ID NCT01665326
First seen Apr 14, 2026 · Last updated May 08, 2026 · Updated 5 times
Summary
This study follows up to 400 people with Pompe disease to see how their immune system reacts to standard enzyme replacement therapy. Researchers will collect medical records from birth to age 18 to understand why some patients develop antibodies that block treatment. No new drugs are being tested; the goal is to learn how to better manage the disease.
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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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