Sarepta Therapeutics develops RNA-targeted and gene therapies for rare diseases, with a focus on Duchenne muscular dystrophy. Its products include EXONDYS 51 and VYONDYS 53, injections that skip specific exons of the dystrophin gene to treat Duchenne in eligible patients. The company also has several other product candidates and collaborations with organizations like F. Hoffman-La Roche, Nationwide Children's Hospital, and Genethon.