A pipeline of innovation and hope
Our product pipeline is the best possible expression of who we are, what we do, and what we believe in. It showcases the many incredible ways we’re working to serve patients in need.
U.S. Expanded Access Policy
At PTC we are committed to providing novel treatment options to patients living with serious diseases. To do this, we conduct clinical trials to assess the safety and efficacy of investigational medicines, which may allow us to obtain the necessary regulatory approvals and provide patients with broader access to these medicines.