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Therapeutic areas

Diseases we’re on a mission to change

Since its inception, PTC has been viewed as an expert in RNA biology. Today, our biotechnology platforms have expanded, but harnessing the most promising scientific approach to target the root cause of a disorder has remained constant.

Clinical Trials at PTC

Developing an array of potential therapies ​

PTC is conducting a wide range of clinical trials in a variety of rare diseases. Interested in enrolling in a clinical trial?