PTC Therapeutics, Inc. is a biopharmaceutical company focused on the discovery and development of orally administered, small-molecule drugs that target post-transcriptional control processes. The Company�s lead product candidate includes ataluren, which is used for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation. Ataluren is in late stage clinical development for the treatment of Duchenne muscular dystrophy caused by nonsense mutations (nmDMD) and cystic fibrosis caused by nonsense mutations (nmCF). Ataluren is orally administered small-molecule compound that targets nonsense mutations. The Company is engaged in the development of ataluren for the treatment of genetic disorders in, which a nonsense mutation is the cause of the disease.