Sarepta Therapeutics, Inc.

This study tests a single dose of SRP-9003 gene therapy in 17 people with limb-girdle muscular dystrophy 2E/R4, a rare genetic muscle-weakening disease. The goal is to see if the therapy can restore a missing protein in muscle cells and improve function. Both walkers and non-walk…

This study tests a gene therapy called SRP-9001 for Duchenne muscular dystrophy, a severe muscle-weakening disease. About 148 males, both those who can still walk and those who cannot, will receive either the therapy or a placebo. The goal is to see if the treatment improves arm …

This study tests whether higher doses of the drug eteplirsen can safely improve muscle function in 160 boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. Participants receive weekly infusions of eteplirsen at different doses to find the best balance…

This study tests a one-time 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 help muscles produce a missing protein (beta-sarcoglycan). B…

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 study follows 205 people with specific types of limb-girdle muscular dystrophy (LGMD) to learn how the disease affects movement and breathing over several years. Participants are observed and tested regularly, but no new treatment is given. The goal is to better understand t…

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

This study follows about 400 people with Duchenne muscular dystrophy who have already received the gene therapy SRP-9001 in a previous clinical trial. No new treatment is given. The goal is to monitor long-term safety and how well the therapy continues to work over time. Particip…