PTC Therapeutics Inc

PTC Therapeutics Inc., founded in 1998 and headquartered in South Plainfield, New Jersey, focuses on the discovery and commercialization of novel medicines in several key areas, including rare diseases, oncology, and gene therapy. Within its rare disease portfolio, PTC has developed Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) and Emflaza™ (deflazacort) also for DMD. Addressing the need for innovative cancer treatments, PTC works on targeted therapies for specific cancer types. In the rapidly progressing field of gene therapy, PTC invests in therapies for various genetic diseases. The company's pipeline includes the gene therapy product Upstaza™ (eladocagene exuparvovec) for AADC deficiency. Additionally, they have entered into a collaboration with the Roche Group and Genentech and consequently contributed to the development of Evrysdi™ (risdiplam) for spinal muscular atrophy (SMA). The company's global presence is marked by the sale of its products and services in various countries, with an especially significant presence in the major markets of North America, Europe, and Latin America. However, the availability of specific products in these countries is subject to change based on regulatory approvals and the progression of PTC's commercialization efforts. (Powered by AI)