New drug shows promise for rare muscle disease

NCT ID NCT05027269

First seen Mar 17, 2026 · Last updated May 13, 2026 · Updated 11 times

Summary

This study tested a new medicine called AOC 1001 in 39 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 the body processes it. Participants received either the drug or a placebo by IV, and were followed for up to 6 months.

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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

Locations

  • Kansas University Medical Center

    Kansas City, Kansas, 66205, United States

  • Ohio State University

    Columbus, Ohio, 43221, United States

  • Stanford University

    Palo Alto, California, 94304, United States

  • University of California Los Angeles

    Los Angeles, California, 90095, United States

  • University of Colorado

    Denver, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

Conditions

Explore the condition pages connected to this study.