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Real-World DMD drug study tracks patient progress over years

NCT ID NCT06606340

First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 31 times

Summary

This study follows 300 people with Duchenne muscular dystrophy who are already taking eteplirsen, golodirsen, or casimersen in regular medical care. Researchers will collect information on movement, breathing, and heart function over time to understand how these treatments work in everyday life. The goal is to learn more about long-term outcomes, not to test a new drug.

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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

  • Arkansas Children's Hospital Research Institute

    Little Rock, Arkansas, 72202, United States

  • Atrium Health Wake Forest University Health Sciences

    Winston-Salem, North Carolina, 27157, United States

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010, United States

  • Columbia University Medical Center - PIN

    New York, New York, 10032, United States

  • Cook Children's Hospital

    Fort Worth, Texas, 76104, United States

  • Duke Lenox Baker Children's Hospital

    Durham, North Carolina, 27705, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21287, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033-0850, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15213, United States

  • UVA Children's Hospital

    Charlottesville, Virginia, 22908, United States

  • University Of Iowa Hospitals And Clinics

    Iowa City, Iowa, 52242, United States

  • University of California Davis Medical Center

    Sacramento, California, 95817, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • Washington University

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.