Sarepta Therapeutics, Inc.

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. The goal was to see if it was safe and could produce dystrophin protein. Only…

This early-stage study tested a single dose of SRP-9004 gene therapy in 4 people with limb girdle muscular dystrophy type 2D/R3, a rare muscle-weakening disease. The main goal was to check safety, but researchers also looked at whether the therapy could increase a missing muscle …

This study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E, a rare genetic muscle-weakening disease. Six participants received a one-time infusion to deliver a working copy of the missing gene. The study was terminated early, so final …

This early-stage study tested a gene therapy (delandistrogene moxeparvovec) in 3 boys with Duchenne muscular dystrophy who had pre-existing antibodies that could block the therapy. To overcome this, the boys first received a procedure called plasmapheresis to remove those antibod…

This study tested a gene therapy called SRP-6004 for people with limb girdle muscular dystrophy type 2B/R2, a rare muscle-weakening disease. The goal was to check if the treatment was safe and could help muscles work better. Only 2 people took part before the study was stopped ea…