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.
Our Science
Leveraging our expertise and cutting-edge biotechnology platforms, we’re urgently turning the latest scientific advances into purposeful treatments
Clinical Trials
We're committed to making progress in rare disease through clinical trial research, each and every moment of every day