PTCT

About PTCT

PTC Therapeutics, Inc. is a science-driven, global biopharmaceutical company. The Company is focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. Its portfolio pipeline includes several commercial products and 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 for rare diseases relating to neurology, metabolism and oncology. The Company has two products, Translarna (ataluren) and Emflaza (deflazacort), for the treatment of Duchenne muscular dystrophy (DMD). It also offers Upstaza, a gene therapy for the treatment of Aromatic L-Amino Decarboxylase (AADC) deficiency, and Evrysdi, a treatment for spinal muscular atrophy (SMA). Its splicing platform includes PTC518, which is being developed for the treatment of Huntington’s disease (HD).