Sarepta Therapeutics, Inc.

This early-stage study aimed to test the safety of a single-dose gene therapy called SRP-9004 for a rare, inherited muscle-wasting disorder. It involved a small group of participants, some who could walk and some who could not, to see if the treatment was safe and if it could inc…

This study tested a two-step treatment for Duchenne muscular dystrophy (DMD). First, a drug (imlifidase) was given to temporarily lower antibodies that might block the gene therapy. Then, the gene therapy (SRP-9001) was given to try to help muscles produce a needed protein. The s…

This early-stage study tested whether a gene therapy called SRP-6004 could safely deliver a working gene to people with limb girdle muscular dystrophy type 2B, a rare genetic muscle disorder. The trial involved just 2 participants who could still walk and had specific genetic mut…

This early-stage study tested a one-time gene therapy for Duchenne muscular dystrophy (DMD) in a very specific group: three boys who already had antibodies that might block the treatment. To try and get around this, doctors first used a blood-filtering procedure called plasmapher…

This was an early-stage study to test the safety and initial effects of an experimental gene therapy called SRP-9003 for people with a rare, inherited muscle-wasting disease called LGMD2E. The therapy aimed to deliver a working copy of a faulty gene directly into the body via an …