Gene therapy showdown: can ELEVIDYS outperform standard care in duchenne?
NCT ID NCT06270719
First seen Nov 01, 2025 · Last updated May 17, 2026 · Updated 31 times
Summary
This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to usual steroid treatment. Researchers will track movement, breathing, and safety over time. Participants must already be on or starting steroids, and those getting gene therapy must be at least 4 years old.
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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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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
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