Sarepta Therapeutics, Inc.

This early-stage study is testing a single dose of an experimental gene therapy called SRP-9003 in six people with a rare genetic muscle disease called limb girdle muscular dystrophy type 2E/R4. The main goals are to check if the treatment is safe and to see if it increases a nee…

This Phase 3 study is testing a single-dose gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E/R4. The therapy aims to deliver a working copy of the beta-sarcoglycan gene to help muscles produce a crucial protein they lack. The study will measure …

This study follows 500 boys and young men with Duchenne muscular dystrophy to compare a new gene therapy (delandistrogene moxeparvovec) against standard steroid treatment in real-world medical settings. Researchers will track walking ability, muscle function, heart health, and sa…

This study is tracking the long-term safety and effectiveness of a gene therapy called delandistrogene moxeparvovec (SRP-9001) in people with Duchenne muscular dystrophy. It will follow 400 participants who have already received the therapy in a previous clinical trial. The goal …

This study is testing whether higher doses of the drug eteplirsen are safe and more effective at slowing muscle decline in boys with Duchenne muscular dystrophy (DMD). It involves about 160 boys aged 4 and older who can still walk and have a specific genetic mutation. The main go…

This study aims to understand how four specific types of limb-girdle muscular dystrophy (LGMD) change over time. It will follow 205 participants, aged 4 and older, for up to 5 years to track their mobility, lung function, and other symptoms. The goal is to gather detailed informa…

This study aims to collect long-term information on how well three specific Duchenne muscular dystrophy (DMD) treatments work in everyday clinical practice. It will follow about 300 boys and young men who are already prescribed these therapies by their doctors. Researchers will t…