Hope for DM1: new drug tested for long-term safety
NCT ID NCT07008469
First seen Nov 01, 2025 · Last updated May 08, 2026 · Updated 30 times
Summary
This study looks at the long-term safety and effects of a drug called AOC 1001 in people with myotonic dystrophy type 1 (DM1), a genetic muscle disease. About 230 adults who already took part in earlier AOC 1001 studies will receive the drug intravenously. The main goal is to track any side effects over time.
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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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AP-HP Hopital Pitie-Salpetriere
Paris, 75013, France
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Aomori Hospital
Aomori, Aomori, 038-1331, Japan
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Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
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Duke University Medical Center
Durham, North Carolina, 27708, United States
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Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milan, 20162, Italy
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Houston Methodist Neurological Institute
Houston, Texas, 77030, United States
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Indiana University (IU)
Indianapolis, Indiana, 46202, United States
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Kansas University Medical Center
Kansas City, Kansas, 66205, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Klinikum der Ludwig-Maximilians-Universitaet Muenchen
München, 80336, Germany
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Montreal Neurological Institute
Montreal, Quebec, H3A 2B4, Canada
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National Hospital Organization Osaka Toneyama Medical Center
Osaka, 560-8552, Japan
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Ohio State University
Columbus, Ohio, 43221, United States
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Osaka University Hospital
Osaka, 565-0871, Japan
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Stanford University
Stanford, California, 94305, United States
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Stichting Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA, Netherlands
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The Ottawa Hospital
Ottawa, Ontario, K1Y 4E9, Canada
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University Research Center of South Florida
Tampa, Florida, 33612, United States
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University of California, Los Angeles (UCLA)
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 Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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University of Washington
Seattle, Washington, 98104, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
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Wake Forest
Winston-Salem, North Carolina, 27157, United States
Conditions
Explore the condition pages connected to this study.