Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, siRNA platform, gene therapy, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping; AMONDYS 45 for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping; and ELEVIDYS, an AAV-based gene therapy, which is contraindicated in patients with any deletion in exon 8 …