FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy 

We’re pleased to share that Sarepta Therapeutics' gene therapy for Duchenne, ELEVIDYS (SRP-9001), has been granted Accelerated Approval by the FDA for individuals with Duchenne ages 4-5. It’s the first approved gene therapy for Duchenne, marking an important milestone that keeps us on a path toward identifying truly transformative treatments for all those living with Duchenne muscular dystrophy. 
Aligned with our mission of driving early and critical Duchenne research, CureDuchenne is proud to have provided early funding to the company which later became Sarepta, helping them move forward and become the company they are today, with three FDA approved exon-skipping drugs for Duchenne and now an approved gene therapy drug for Duchenne.  
While this marks important progress, we recognize this is just a first step, and our work is far from done. There are still many individuals waiting for treatments, and we will continue to support and invest in promising research to bring solutions to everyone in need.
Learn more about CureDuchenne at cureduchenne.org