At PTC, we dedicate ourselves to using groundbreaking science as we research rare diseases. We believe that by shifting our perspective, using the newest technologies available, we can find innovative ways to treat these diseases and create shared moments between patients and their families. Coupled with our patient-centric approach to treatment, we create an environment that resembles more of a family get-together than a typical doctor-patient relationship. In doing all of this, we can change lives around the world.
The Science Behind Our Treatment Pipeline
We commit ourselves to pushing the envelope of what is possible, whether in a niche oncology platform, gene therapy, or an emerging treatment. However, we also seek to expand our internal treatment pipeline by analyzing our scientists’ research and collaborating with other like-minded groups. All of this aids us in our mission to produce commercial products and medications that can help those with ultra-orphan (aka very rare) disorders enjoy more moments with those they love.