An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice. In addition, treatment outcomes will be collected prospectively from post-trial participants who have received delandistrogene moxeparvovec through participation in select SRP-9001 studies.
- EligibilityCriteria: Inclusion Criteria:  - Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.  Among participants recruited from routine clinical practice:  Is aged 4 through 5 years at the time of enrollment. Is ambulatory per protocol specified criteria. Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.  For Delandistrogene Moxeparvovec-exposed Participants:  - Will be initiating usual care treatment with delandistrogene moxeparvovec at the time of study enrollment.  For Comparators:  - Is unexposed to DMD gene therapy at the time of study enrollment.  Exclusion Criteria:  Among participants recruited from routine clinical practice:  Has any deletion of exon 8 and/or exon 9 in the DMD gene. Is currently participating in any DMD interventional study at the time of this observational study enrollment.  Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:  The participant's ability to comply with the protocol-required procedures, The participant's wellbeing or safety, and/or The clinical interpretability of the data collected from the participant.  Other inclusion/exclusion criteria may apply. - HealthyVolunteers: No - Gender: Male - MinimumAge: 4 Years - StdAgeList: Child, Adult, Older Adult - StudyPopulation: DMD participants within each prescriber's practice who either (1) have been prescribed delandistrogene moxeparvovec or (2) are receiving or have been prescribed to begin chronic glucocorticoid therapy in routine clinical practice, and who meet the study eligibility criteria, will be invited to enroll into the study and will be followed according to the protocol. - SamplingMethod: Non-Probability Sample