Tracking hope: Real-World study follows boys with DMD on specialized therapies

NCT ID NCT06606340

Summary

This study aims to collect long-term information on how well three specific Duchenne muscular dystrophy (DMD) treatments work in everyday clinical practice. It will follow about 300 boys and young men who are already prescribed these therapies by their doctors. Researchers will track key physical abilities like walking, standing up, lung function, and heart health over time to understand the real-world benefits and progression of the disease while on treatment.

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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.