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
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Locations
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Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, 72202, United States
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Atrium Health Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
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Columbia University Medical Center - PIN
New York, New York, 10032, 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, 27705, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21287, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850, United States
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Seattle Children's Hospital
Seattle, Washington, 98105, United States
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Texas Children's Hospital
Houston, Texas, 77030, United States
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UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213, United States
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UVA Children's Hospital
Charlottesville, Virginia, 22908, United States
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University Of Iowa Hospitals And Clinics
Iowa City, Iowa, 52242, United States
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University of California Davis Medical Center
Sacramento, California, 95817, United States
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University of Florida
Gainesville, Florida, 32610, United States
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Washington University
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.