New pompe disease drug enters early human testing
NCT ID NCT06109948
First seen Jan 06, 2026 · Last updated May 02, 2026 · Updated 17 times
Summary
This early-phase study tests a new drug called ABX1100 in healthy volunteers and people with late-onset Pompe disease. The main goal is to check if the drug is safe and how the body processes it. The study is small, with 46 participants, and is not yet testing if the drug works as a treatment.
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This is a summary of
the original study
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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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Lysosomal and Rare Disorders Research and Treatment Center, Inc
Fairfax, Virginia, 22030, United States
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MAGIC clinic
Calgary, Alberta, Canada
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McMaster University
Hamilton, Ontario, Canada
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UCI
Orange, California, 92868, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
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