PTC Therapeutics, Inc. is a global biopharmaceutical company. The Company is focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. Its diversified therapeutic portfolio includes several commercial products and product candidates in various stages of development, including discovery, research and clinical stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases relating to neurology and metabolism. It has two products, Translarna (ataluren) and Emflaza (deflazacort), for the treatment of Duchenne muscular dystrophy (DMD) a rare, life-threatening disorder. Its Upstaza, a gene therapy for the treatment of Aromatic L-Amino Decarboxylase (AADC) deficiency, a rare central nervous system (CNS) disorder. Its Tegsedi and Waylivra are for the treatment of rare diseases. Its Evrysdi, a treatment for spinal muscular atrophy (SMA).