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
We are committed to making progress in rare disease through clinical trial research.
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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NEWSROOM
Andrea’s Message to the FA Community
Andrea lives with Friedreich’s ataxia (FA), a rare, genetic, and progressive neuromuscular disease that mainly affects the central nervous system and the heart. She has shared her story with PTC to help spread awareness about FA. -
PRESS RELEASE
PTC Therapeutics Provides Corporate Update and Reports First Quarter 2025 Financial Results
PTC Therapeutics announced a corporate update and financial results for the first quarter ended March 31, 2025. Highligt include a promising start thanks to strong revenue performance and a steady progression of global launch activities following the recent positive CHMP opinion for Sephience. -
PRESS RELEASE
PTC518 PIVOT-HD Study Achieves Primary Endpoint
PTC Therapeutics announced results from its Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients. The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p<0.0001) at Week 12 and favorable safety and tolerability.
PTC works closely with healthcare providers to support their needs
Support for the PTC patient, family, and caregiver community
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