A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

To join this study, participants must have Limb-girdle Muscular Dystrophy type 2E/R4 (LGMD2E/R4) and specific genetic changes in the beta-sarcoglycan gene. There are two main groups being looked for: those who can walk independently and those who cannot.

If you can walk, you would need to be able to walk a certain distance within a specific time and have good scores on some activity tests. If you cannot walk, there are different criteria related to how quickly you can walk that distance, or if you can't walk it at all, and your scores on other activity measures.

There are also some other important checks. For example, your body shouldn't have too many antibodies to a certain virus (AAVrh74). Also, you wouldn't be able to join if you have serious heart or lung problems, an autoimmune disease that needs active treatment, or any other major health issues besides your LGMD2E/R4.

If you decide to take part in this study, you would receive a single dose of the experimental gene therapy, SRP-9003. You would also receive a type of medicine called a glucocorticoid, which helps manage the body's reaction to the gene therapy. Throughout the study, you would attend regular appointments at a clinic where doctors and nurses would perform various tests. These tests might include muscle strength assessments, walking tests, blood tests, and potentially others to see how the treatment is affecting you and your condition. The study duration isn't specified, but clinical trials typically involve several follow-up visits over months or even years to monitor effects and safety.