PTC Therapeutics is a global biopharmaceutical company founded in 1998, dedicated to the discovery and development of innovative treatments for rare diseases. Initially focused on orally administered small molecule drugs targeting post-transcriptional control processes, PTC has expanded its technology platforms to include gene therapy and various approaches to address multiple therapeutic areas. The company is particularly committed to developing and commercializing treatments for orphan and ultra-orphan disorders, with a pipeline that includes therapies for neuromuscular disorders, oncology, and metabolic diseases. PTC's mission is to provide access to best-in-class treatments for patients with rare diseases who have limited or no treatment options, emphasizing a cause-driven approach to healthcare.