A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.'s Exon-Skipping Therapies in Patients With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

Sarepta Therapeutics, Inc.

Summary

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.

Eligibility

Age range: Not specified
Sex: Male
Healthy volunteers: No

Detailed criteria

Key Inclusion Criteria: * Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures. * Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report. * Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen…

Interventions

Drug
Eteplirsen
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.
Drug
Golodirsen
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.
Drug
Casimersen
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.

Locations (20)

Arkansas Children's Hospital Research Institute
Little Rock, Arkansas
Children's Hospital Los Angeles
Los Angeles, California
University of California Davis Medical Center
Sacramento, California
Children's National Hospital
Washington D.C., District of Columbia
University of Florida
Gainesville, Florida
University Of Iowa Hospitals And Clinics
Iowa City, Iowa