Upon joining the study, the patient must have a signed informed consent form. If applicable, a signed assent form is also required, depending on the patient’s age and local standards.

The patient must be male and meet specific age requirements for the cohort they are assigned to. For example, Cohort A requires the patient to be 3 years old, while Cohort D requires the patient to be 6 months old or younger.

A definitive diagnosis of Duchenne Muscular Dystrophy (DMD) is necessary, confirmed by genetic testing showing specific mutations in the DMD gene.

The patient undergoes an initial assessment to confirm eligibility, including the ability to cooperate with motor assessment testing.

The patient’s parent or legal guardian must be able to understand and comply with the study visit schedule and protocol requirements.

The patient receives the study medication, delandistrogene moxeparvovec-rokl, which is a solution for injection or infusion.

The medication is administered intravenously, meaning it is given directly into a vein.

The patient is monitored for any treatment-emergent adverse events, serious adverse events, and adverse events of special interest.

Regular assessments are conducted to check for clinically significant changes in vital signs, physical examination findings, safety laboratory assessments, ECGs, and ECHOs.

The study also measures changes in the quantity of SRP-9001-dystrophin protein expression from baseline to Week 12 using a method called Western blot.

The study is estimated to continue until November 30, 2032.

The recruitment for the study began on July 1, 2023.