Profile

PTC Therapeutics is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control mechanisms in orphan diseases. In September 2009, PTC entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases. In 2020, PTC announced the FDA approval of Evrysdi™ for the treatment of spinal muscular atrophy in adults and children 2 months and older, in partnership with the SMA Foundation and Roche.