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Why We Must Consider the ‘Totality of Evidence’ for Novel Rare Disease Therapies

May 15, 2023

  1. Why We Must Consider the ‘Totality of Evidence’ for Novel Rare Disease Therapies

    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…

  2. What’s So Special About 2023?

    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…

  3. 2022: A Year of Many Firsts

    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…

  4. BioTech Breakthrough Awards: PTC is 2022 BioTechnology Innovator of the Year

    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…

  5. PTC is a Finalist in Two Pharma Industry Awards in Ireland

    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…

  6. An innovative supply chain model for an innovative product

    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…

  7. Preparing Europe for a new generation of innovative therapies

    Thomas Bols, Head of Government Affairs and Patient Advocacy, EMEA and APAC at PTC, recently shared his perspective with Open Access Government on how preparing Europe for a new generation of innovative therapies has the potential to transform the lives of patients. Gene therapies are bringing hope to patients living with ultra-rare diseases. In the…

  8. PTC joins Screen4Care to shorten the diagnosis journey for people living with rare diseases

    For too many people living with rare diseases, getting a correct diagnosis is a lengthy process that negatively impacts patients and their family. Through an innovative research collaboration called Screen4Care, PTC will be working alongside 36 international partners from academia, industry, and patient advocacy organizations to find solutions to shorten the path to diagnosis and…

  9. Career Opportunity: Executive Director, Global Medical Affairs – Global Gene Therapy Lead

    Join the Global Medical Affairs Team and Advance our Gene Therapy Business At PTC Therapeutics, our science-driven #OnePTC team combines our strong clinical and scientific expertise with dedication to using groundbreaking science and new technologies to find innovative ways to treat diseases with high unmet need. Our Global Medical Affairs team drives this mission forward…

  10. What’s in Store for PTC in 2022

    Last week, we presented at the 40th Annual J.P. Morgan Healthcare Conference, an annual healthcare investment symposium connecting healthcare industry leaders, innovators, and the broader healthcare industry. Even though this year’s meeting was (once again) virtual, the excitement for everything that’s to come in 2022 was palpable – especially for us at PTC, as 2022…