Status
Conditions
Treatments
About
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.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
A participant recruited to Cohorts 1a or 2:
A participant recruited to Cohort 1b:
For Delandistrogene Moxeparvovec-treated Participants:
For Comparators:
Exclusion criteria
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:
Other inclusion/exclusion criteria may apply.
500 participants in 2 patient groups
Loading...
Central trial contact
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal