PTC Therapeutics

PTC Therapeutics is a global biopharmaceutical company that discovers, develops and commercializes medicines for children and adults living with rare disorders. Its work draws on its scientific expertise in post-transcriptional regulation and its splicing and other small-molecule platforms. Its marketed products include Emflaza (deflazacort) for Duchenne muscular dystrophy, Translarna (ataluren) in certain regions, Evrysdi (risdiplam, with Roche and Genentech) for spinal muscular atrophy, Kebilidi/Upstaza (eladocagene exuparvovec) for AADC deficiency, and Sephience (sepiapterin) for phenylketonuria.