Transforming Lives
Innovation, fueled by science and a passion for patients
PTC is an established global biopharmaceutical company that delivers transformative therapies for people living with rare diseases. For 25 years, we have been harnessing our scientific platforms to create new therapies that address the underlying cause of the disease and deliver on our promise to create more moments for those who count on us.
Focused research platforms at work for patients
Challenging what's possible
Harnessing groundbreaking science for patients has led to many firsts – the first approved treatment for Duchenne in the world, the first approved gene therapy directly infused into the brain, and the first approved small molecule splicing modifier. Our innovative pipeline continues to expand as we discover and develop treatments using the newest technologies available.
Clinical Trials
Click the links below to learn more about our actively enrolling trials
We love what we do
PTC employees are working to change patients’ lives every single day.
We are motivated by our patients and the drive for them to have more moments with their families, made possible in part by our research and therapies.
If you’re looking to take on rewarding work with inspiring colleagues, come grow with us.
Making headlines
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PRESS RELEASE
PTC Announces Submission of Sepiapterin MAA for Treatment of PKU to EMA
First marketing authorization submission for sepiapterin with additional global submissions to follow in 2024 -
NEWSROOM
What You Need to Know About Ferroptosis
Our research efforts at PTC are focused on two scientific platforms – splicing and ferroptosis and inflammation – where PTC has unique expertise to discover and advance innovative therapies to the clinic. -
PRESS RELEASE
PTC Therapeutics Provides Key Regulatory Updates
BLA submitted to FDA for Upstaza™ for the treatment of AADC deficiency and NDA to be submitted mid-year for Translarna™ based on FDA feedback.