This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.
Study Type
OBSERVATIONAL
Enrollment
300
No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.
No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.
No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, United States
Loss of Ambulation (LOA)
Time frame: Up to 5 years
Time to Rise From the Floor (Supine to Stand)
Time frame: Up to 5 years
Time to Walk/Run 10 Meters
Time frame: Up to 5 years
Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A
Time frame: Up to 5 years
Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)
Time frame: Up to 5 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
Time frame: Up to 5 years
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Children's Hospital Los Angeles
Los Angeles, California, United States
University of California Davis Medical Center
Sacramento, California, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
University of Florida
Gainesville, Florida, United States
University Of Iowa Hospitals And Clinics
Iowa City, Iowa, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Washington University
St Louis, Missouri, United States
Columbia University Medical Center - PIN
New York, New York, United States
Duke Lenox Baker Children's Hospital
Durham, North Carolina, United States
...and 10 more locations