This is an observational study that follows, over the long term, participants who previously received the RGX-202 gene therapy as a single infusion for Duchenne muscular dystrophy (DMD). No new treatment is given; only people who took part in earlier studies are followed. It is a follow-up study with no assigned phase and enrolls by invitation.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * The parent(s) or legal guardian(s) of the participant has/(have) provided written informed consent and (where applicable) Health Insurance Portability and Accountability Act (HIPAA) authorization after the nature of the study has been explained, prior to any research-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements. * Must have undergone evaluation in a previous clinical study following a single IV infusion of RGX-202 for the treatment of DMD and either completed or withdrawn early from that study. * Participant and parent(s)/legal guardian(s) are willing and able to comply with scheduled visits, and study procedures. * Sexually active participants must be willing to use a medically accepted method of contraception from the time of the screening visit through 5 years after RGX-202 administration. Exclusion Criteria: * No exclusion criteria apply in this observational follow up study.