First patients test new genetic drug for rare muscle disease
NCT ID NCT06109948
Summary
This is the first human study testing ABX1100, a new drug for late-onset Pompe disease—a rare genetic disorder that weakens muscles. Researchers will check the safety of different doses in 46 healthy volunteers and patients with the disease. The study will measure how the drug moves through the body and its effects on muscle tissue.
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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
Explore the condition pages connected to this study.