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Our Science

Pioneering Innovation

Our research is centered on two scientific platforms—Splicing and Inflammation/ferroptosis

We have focused our research efforts on two scientific platforms—splicing and inflammation/ferroptosis — where PTC has unique expertise to discover and advance to the clinic innovative therapies.

Our Scientific Platforms

Splicing

Modulating splicing to control protein production

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Inflammation/ferroptosis

Targeting oxidative stress and inflammation pathways

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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.

Explore our Products

Read More About Science and Innovation at PTC

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International Clinical Trials Day: Patients Lead the Way

On May 20, we recognize International Clinical Trials Day: a time to honor everyone who makes clinical research possible, especially the…

What You Need to Know About Ferroptosis & Friedreich’s Ataxia (FA)

An Informative Q&A with Jonathan Cherry, Global Project Leader Our research efforts at PTC are focused on two scientific platforms…

What You Need to Know About Ferroptosis

Our research efforts at PTC are focused on two scientific platforms – splicing and inflammation/ferroptosis – where PTC has unique…

A Whole Team in One Molecule: PTC’s Splicing Platform

There are many things that make PTC Therapeutics truly unique, and the splicing platform is one of them. Here, “splicing” refers to…