PTC Therapeutics is a biopharmaceutical company focused on the discovery, development, and commercialization of orally-administered, small molecule therapeutics that focus on post-transcriptional control processes. The Company's lead product, Translarna, is used for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients with age of over five years and older. Its programs include Translarna for nonsense mutation cystic fibrosis, Translarna for mucopolysaccharidosis type I caused by nonsense mutation, Translarna for nonsense mutation aniridia, and Translarna for nonsense mutation Dravet syndrome/CDKL5, Spinal muscular atrophy in collaboration with Spinal Muscular Atrophy Foundation.