New hope for pompe? early trial tests ABX1100
NCT ID NCT06109948
First seen Jan 06, 2026 · Last updated May 26, 2026 · Updated 19 times
Summary
This early-phase study tested a new drug called ABX1100 in 46 people, including healthy volunteers and patients with late-onset Pompe disease. The main goal was to check safety and how the drug moves through the body. Researchers gave single or multiple doses to see if it's safe enough for further testing.
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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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