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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eteplirsen |
| ||
| Golodirsen |
| ||
| Casimersen |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eteplirsen | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Loss of Ambulation (LOA) | Up to 5 years | |
| Time to Rise From the Floor (Supine to Stand) | Up to 5 years | |
| Time to Walk/Run 10 Meters | Up to 5 years | |
| Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A | Up to 5 years | |
| Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted) | Up to 5 years | |
| Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO) | Up to 5 years |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Is currently participating in any DMD interventional study at the time of this study enrollment.
Has declined to provide consent for collection of their genetic data.
Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise:
Other inclusion/exclusion criteria may apply.
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Participants within each prescriber's practice who are receiving eteplirsen, golodirsen, or casimersen, in routine clinical practice, and who meet the study eligibility criteria and provide informed consent (either by the participant or through authorization by a legal guardian), will be invited to enroll into the study and will be followed according to the protocol.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital Research Institute | Little Rock | Arkansas | 72202 | United States | ||
| Children's Hospital Los Angeles |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41961051 | Derived | Tian C, Grabich S, Veerapandiyan A, Scharf RJ, Santra S, Hornibrook S, Drummond K, Sehinovych I, Waldrop M. Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD. J Comp Eff Res. 2026 May;15(5):e250108. doi: 10.57264/cer-2025-0108. Epub 2026 Apr 10. |
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| Golodirsen | Drug | 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. |
|
|
| Casimersen | Drug | 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. |
|
|
| Los Angeles |
| California |
| 90027 |
| United States |
| University of California Davis Medical Center | Sacramento | California | 95817 | United States |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | United States |
| University of Florida | Gainesville | Florida | 32610 | United States |
| University Of Iowa Hospitals And Clinics | Iowa City | Iowa | 52242 | United States |
| Kennedy Krieger Institute | Baltimore | Maryland | 21287 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Columbia University Medical Center - PIN | New York | New York | 10032 | United States |
| Duke Lenox Baker Children's Hospital | Durham | North Carolina | 27705 | United States |
| Atrium Health Wake Forest University Health Sciences | Winston-Salem | North Carolina | 27157 | United States |
| Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | 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 |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15213 | United States |
| Cook Children's Hospital | Fort Worth | Texas | 76104 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| UVA Children's Hospital | Charlottesville | Virginia | 22908 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000611335 | eteplirsen |
| C000710673 | golodirsen |
| C000718147 | casimersen |
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