New drug shows promise in early trial for rare muscle disease
NCT ID NCT05027269
First seen Mar 17, 2026 · Last updated May 20, 2026 · Updated 12 times
Summary
This study tested a new medicine called AOC 1001 in 38 adults with myotonic dystrophy type 1 (DM1), a genetic disease that causes muscle weakness and stiffness. The goal was to see if the drug is safe and how it works in the body. Participants received either the drug or a placebo by IV, and were followed for up to 6 months. The results help researchers understand the right dose and any side effects, paving the way for larger studies.
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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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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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