公司简介
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; ...
关联新闻
- Dyne, Sarepta shares tumble as Novartis setback weighs on muscle disease drug developers
Shares of Dyne Therapeutics and Sarepta slumped on Tuesday after Novartis' treatment for a muscle-wasting disorder failed in a trial, fueling investor concerns about the prospects of similar therapie...