First human trial tests potential new treatment for rare muscle disease
NCT ID NCT05027269
Summary
This study tested the safety and effects of a new drug called AOC 1001 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. Thirty-nine participants received either the drug or a placebo through an IV to see how their bodies handled it and if it affected the disease. The main goal was to check for side effects and understand how the drug moves through the body.
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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Kansas University Medical Center
Kansas City, Kansas, 66205, United States
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Ohio State University
Columbus, Ohio, 43221, United States
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Stanford University
Palo Alto, California, 94304, United States
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University of California Los Angeles
Los Angeles, California, 90095, United States
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University of Colorado
Denver, Colorado, 80045, United States
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University of Florida
Gainesville, Florida, 32608, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Conditions
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