Diseases we’re on a mission to change
We are deeply committed to patients living with rare diseases who have little-to-no treatment options, and this passion fuels and inspires us. Our commercial and development efforts are focused in two therapeutic areas – neurology and metabolic conditions – in which we have deep experience and a record of success.
Since our founding in 1998, PTC has concentrated primarily on post-translational control mechanisms in orphan diseases. As we have innovated and grown, our area of therapeutic focus has expanded from this foundational scientific expertise in RNA mechanisms. As in decades past, we continue to work toward innovative therapies for rare diseases.
We are currently working on developing therapies for neurological and metabolic disorders.
Clinical Trials at PTC
PTC is conducting clinical trials in a number of different rare diseases.
Details of on-going and completed phase I to IV clinical trials run by PTC are available to view on: www.clinicaltrials.gov
Results of PTC trials are published on www.clinicaltrials.gov and in peer reviewed journals.