The latest news from PTC

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. We recently sat down with Jeff Trimmer, Senior VP, Research Site Head, to learn more about ferroptosis, how it relates to inflammation and…

There are many things that make PTC Therapeutics truly unique, and the splicing platform is one of them. Here, “splicing” refers to PTC’s scientific platform that takes aim at modulating pre-messenger RNA (pre-mRNA) splicing to control protein production. This scientific platform together with the technology that powers it, enables the identification of small molecules, which…

It’s Calum Sinclair’s job to help ensure patient safety. As the Vice President, Global Pharmacovigilance (PV) at PTC, Calum leads a global team who collect, assess, and monitor the use and safety of PTC’s medicines, from clinical to commercial setting.   It’s a highly important role that Calum candidly enjoys and has throughout his extensive career…

By Rafael Sierra, Executive Director, Global Medical Affairs – Global Gene Therapy Lead Rare and ultra rare diseases present unique challenges for research and healthcare, especially when patients are geographically dispersed, and the condition presents with high heterogeneity. Disease registries have emerged as a valuable tool to continue understanding the disease progression and the natural…

Judit Illes, Director, Government Affairs & Public Policy at PTC recently contributed to a newly published white paper, “Pioneering the New Era of Newborn Screening.” We connected with Judit to learn more about newborn screening, why it is so important and what she hopes people take away from this comprehensive paper on proposed reforms to…

By Jonathan Blaize, PhD, Medical Science Liaison, Medical Affairs, PTC To promote awareness of common behavioral concerns, provide a platform for scientific exchange amongst clinicians and to identify best practices for the management of patients with Duchenne muscular dystrophy (DMD), PTC partnered with Dr. Natalie Truba in 2020 to create a colloquium series focused on…

PTC Italy recently won two distinct industry awards in innovation, leadership and patient-centricity: the Le Fonti Award for Excellence in Biopharmaceutical Innovation & Leadership of the Year and The Excellence of Scientific Information and the Centrality of the Patient Award. At the thirteenth edition of the Le Fonti Awards, PTC was honored to accept the…

At the Life Sciences Industry Awards in Ireland on July 6, PTC received an award for Supply Chain Achievement and was named a finalist in the Life Sciences Company of the Year SME award. The Life Science Industry Awards recognize and celebrate excellence in the Irish life science industry. PTC teams in Ireland were proud…

Neil Smith, Global PKU Project Leader for PTC Therapeutics, has always been driven to improve the lives of other people. Originally trained as a pharmacist, Neil has worked for more than two decades in the pharmaceutical industry, stepping into various roles at both large pharmaceutical companies and small biotech firms, and building a diverse skill…

Christian Werner, Executive Director, Global Medical Affairs, Global DMD Lead, PTC, believes the “totality of evidence” provides an important understanding of the impact of a unique treatment. In this article, he shares strategies for broadening the adoption of the totality of evidence. As a physician working in rare disease drug development, I am convinced that…