PTC Therapeutics, Inc. operates as a fully integrated biopharmaceutical company with a global footprint spanning over 50 countries. The company's core scientific approach leverages its expertise in RNA biology and post-transcriptional control processes to develop small-molecule drugs and gene therapies. Key commercial products include treatments for Duchenne muscular dystrophy (DMD), such as Translarna and Emflaza, as well as Evrysdi for spinal muscular atrophy (SMA), which was developed in partnership with Roche. Additionally, PTC has introduced Upstaza, a pioneering gene therapy for AADC deficiency, and Sephience for phenylketonuria (PKU). The company generates revenue through direct product sales and royalty agreements, maintaining a robust pipeline of clinical candidates for rare genetic and metabolic disorders.
