Sarepta Therapeutics, Inc.

This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to usual steroid treatment. Researchers will track movement, breathing, and safety over time. Participants must already be on or starting steroids, and those ge…

This early-stage study tests a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can restore a missing muscle protein. Both walker…

This study tests whether higher doses of the drug eteplirsen can safely help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. About 160 boys who can still walk will receive different doses to see if it improves muscle strength and daily movement. …

This study tests a single dose of a gene therapy called SRP-9003 in 17 people with limb-girdle muscular dystrophy 2E/R4, a rare muscle-weakening disease. The treatment delivers a working copy of the faulty gene to muscle cells. Researchers will measure if the therapy increases th…

This study follows 300 people with Duchenne muscular dystrophy who are already taking eteplirsen, golodirsen, or casimersen in everyday medical care. Researchers will collect information on movement, breathing, and heart function over time to see how these treatments work in the …

This study follows 205 people with certain types of limb-girdle muscular dystrophy (LGMD) to learn how the disease changes over time. Researchers measure muscle strength, walking ability, and lung function for up to 5 years. The goal is to better understand the disease, which may…

This study tracks 400 people with Duchenne muscular dystrophy who previously received the gene therapy SRP-9001. Researchers will monitor their health and muscle function over time to understand long-term safety and effectiveness. No new treatment is given in this study.