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Update to the U.S. Duchenne Community

Our Science Pioneering Innovation

Our research is centered on two scientific platforms—Splicing and Inflammation and Ferroptosis.

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.​

Our Scientific Platforms
Splicing
Modulating splicing to control protein production
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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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Read More About Science and Innovation at PTC

Ferroptosis Q & A
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…
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Ferroptosis and Inflammation
What You Need to Know About Ferroptosis
Our research efforts at PTC are focused on two scientific platforms – splicing and inflammation and ferroptosis – where PTC has unique…
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PTC's Splicing Platform
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…
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Calum Sinclair
An Important Job and Rewarding Career
It’s Calum Sinclair’s job to help ensure patient safety. As the Vice President, Global Pharmacovigilance (PV) at PTC, Calum leads a…
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Our Products

Leveraging our unique scientific knowledge to develop transformative therapies

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Approved Medicines

See the treatments we’re bringing to more patients around the globe

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Therapeutic Areas

Providing for patients living with rare diseases who have little-to-no treatment options

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