Gene therapy vs. steroids: Real-World duchenne battle
NCT ID NCT06270719
Summary
This study follows 500 boys and young men with Duchenne muscular dystrophy to compare a new gene therapy (delandistrogene moxeparvovec) against standard steroid treatment in real-world medical settings. Researchers will track walking ability, muscle function, heart health, and safety over time to see which approach works better. The study includes both walking and non-walking participants to understand how the treatments affect different stages of the disease.
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Contacts and locations
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Locations
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All Children's Research Institute, Inc
St. Petersburg, Florida, 33701, United States
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Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611-2991, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Children's Hospital Los Angeles - PIN
Los Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Childrens Hospital Medical Center of Akron
Akron, Ohio, 44302, United States
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Connecticut Children's Medical Center - Hartford
Hartford, Connecticut, 06106, United States
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Cook Children's Hospital
Fort Worth, Texas, 76104, United States
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Duke Lenox Baker Children's Hospital
Durham, North Carolina, 27704, United States
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Indiana Clinical and Translational Science Institute
Indianapolis, Indiana, 46202, United States
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Le Bonheur Children's Hospital
Memphis, Tennessee, 38103, United States
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Nemours Children's Hospital - Orlando
Orlando, Florida, 32827, United States
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Nicklaus Children's Hospital
Miami, Florida, 33155, United States
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OHSU Healthcare (Oregon Health and Science University)
Portland, Oregon, 97239, United States
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Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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The Board of Regents of the University of Wisconsin
Madison, Wisconsin, 53715-1218, United States
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The Medical College of Wisconsin
Milwaukee, Wisconsin, 53226-4874, United States
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University of Colorado - PPDS
Aurora, Colorado, 80045, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Virginia
Charlottesville, Virginia, 22903, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
Conditions
Explore the condition pages connected to this study.