Our science
Pushing the boundaries of innovation
We have focused our research efforts on two scientific platforms—splicing and ferroptosis and inflammation— where PTC has unique expertise to discover and advance to the clinic innovative therapies.
Focused research platforms at work for patients
Ferroptosis & Inflammation
Targeting oxidative stress and inflammation pathways to treat CNS diseases
Learn more
Our Pipeline
Leveraging our unique scientific knowledge
We have a robust development portfolio with a number of potentially promising therapies for rare neurologic and metabolic conditions.
Read More About Science and Innovation at PTC
Our Pipeline
Leveraging our unique scientific knowledge to develop transformative therapies
Therapeutic Areas
Providing for patients living with rare diseases who have little-to-no treatment options