The latest news from PTC

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…

2023 is a meaningful, unique and thrilling year for PTC. It’s the company’s 25th anniversary, and it is poised to be a year of momentum as we anticipate significant trial readouts in multiple disease areas. We’re also working toward our ambitious goal to produce a therapy every two to three years based on our robust…

For PTC, 2022 was a milestone-reaching, patient-supporting, award-winning, employee-engaging, community-driven year. It was also another year of many firsts. The first approved disease-modifying treatment for AADC deficiency, and the first marketed gene therapy directly infused into the brain, was approved in Europe and the UK. We also had the first global regulatory approval for treatment…

PTC has been selected as “BioTechnology Innovator of the Year” in the second annual BioTech Breakthrough Awards program conducted by BioTech Breakthrough, a leading independent market intelligence organization that evaluates and recognizes standout life sciences and biotechnology companies, products and services around the globe. The award comes in recognition of our use of pioneering science…

PTC was thrilled to be shortlisted as a finalist in the Pharma Industry Awards in two categories, Biotech Company of the Year and Supply Chain Achievement Award, alongside remarkable companies and organizations in Ireland’s pharma industry. The Pharma Industry Awards recognize and celebrate the most original and innovative individuals and companies that demonstrate excellence in…

In July 2022, PTC’s first gene therapy and disease-modifying treatment for Aromatic l‐amino acid decarboxylase (AADC) deficiency was approved by the European Commission (EC). In this relatively new field of molecular medicine, PTC is one of the pioneers in the gene therapy, being the first therapy to be injected directly into the brain. Unlike the…