Sarepta Therapeutics, Inc. is a biopharmaceutical company. The Company focuses on the discovery and development of ribose nucleic acid (RNA)-targeted therapeutics for the treatment of rare neuromuscular diseases. It operates through discovering, developing, manufacturing and delivering therapies to patients with Duchenne muscular dystrophy (DMD). It is focused on the development of its disease-modifying DMD drug candidates. It has received accelerated approval for its product, EXONDYS 51, indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. EXONDYS 51 is studied in clinical trials under the name of eteplirsen. Its next generation phosphorodiamidate morpholino oligomer (PMO)-based compounds are synthetic compounds that bind to complementary sequences of RNA by standard Watson-Crick nucleobase pairing. Its PMO-based chemistries are peptide conjugated PMO (PPMO), PMO-X and PMOplus.