PTCT

PTC Therapeutics, Inc. is a science-driven global biopharmaceutical company. The Company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. The Company have a portfolio pipeline that includes commercial products as well as product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focused on the development of new treatments for multiple therapeutic areas, including rare diseases and oncology. The Company have two products, Translarna (ataluren) and Emflaza (deflazacort), for the treatment of Duchenne muscular dystrophy (DMD). The Company have a pipeline of gene therapy product candidates for rare monogenic diseases that affect the central nervous system (CNS), including PTC-AADC for the treatment of Aromatic L-Amino Acid Decarboxylase (AADC) and CNSA-001 for phenylketonuria (PKU) diseases.