PTC Therapeutics is a global biopharmaceutical company that discovers, develops, and markets medicines for rare genetic diseases. It uses RNA biology and post-transcriptional control to create therapies, including small-molecule drugs and gene therapies. Its products include Translarna for nmDMD, Evrysdi for SMA, Emflaza, Upstaza for AADC deficiency, and Kebilidi for CNS indications, all supported by an integrated model from research to commercialization and operations in more than 50 countries with a European base in Dublin. The company aims to provide safe, effective treatments to patients with few or no options by building a strong pipeline and broad global access.
Company Size
1,001-5,000
Company Stage
IPO
Headquarters
Warren Township, New Jersey
Founded
1998
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