Our science
Pushing the boundaries of innovation
We have focused our research efforts on two scientific platforms—splicing and inflammation and ferroptosis — where PTC has unique expertise to discover and advance to the clinic innovative therapies.
Focused research platforms at work for patients
Inflammation & Ferroptosis
Targeting oxidative stress and inflammation pathways to treat CNS diseases
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Our Products
Leveraging our unique scientific knowledge
We have a number of potentially promising therapies for rare neurologic and metabolic conditions.
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Our Products
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