Skip to main content

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.
PTC Pipeline - January 2024
PKU, phenylketonuria; FA, Friedreich ataxia; ALS, amyotrophic lateral sclerosis; HD, Huntington‘s disease; SCA-3, spinocerebellar ataxia type 3; MAP-tau, microtubule associated protein tau.

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

Approved Medicines
Approved Medicines

See the treatments we’re bringing to more patients around the globe

Clinical Trials
Clinical Trials

We're committed to making progress in rare disease through clinical trial research, each and every moment of every day