Sarepta Therapeutics, Inc.

This early-phase study tested a single dose of a gene therapy called SRP-9004 in 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, and researchers also looked at changes in muscle protein levels and functi…

This study tested a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a disease that weakens muscles over time. The goal was to see if the treatment was safe when given through a vein. Only 2 people took part before the study was stopped earl…

This study tested a gene therapy called delandistrogene moxeparvovec in 3 boys with Duchenne muscular dystrophy who had antibodies that could block the treatment. Before receiving the therapy, participants underwent a procedure called plasmapheresis to remove those antibodies. Th…

This study tested a gene therapy (delandistrogene moxeparvovec) combined with a drug called imlifidase to help patients with Duchenne muscular dystrophy who have antibodies that could block the therapy. Only 5 participants were planned, and the study was terminated early. The goa…

This early-stage study tested a gene therapy called SRP-9003 in 6 people with Limb-Girdle Muscular Dystrophy Type 2E, a rare genetic condition that causes muscle weakness. The goal was to see if the treatment was safe and could help muscles work better. The study was stopped earl…