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Pioneering Treatments for Rare Diseases: PTC Honors Rare Disease Day 2023

February 23, 2023

  1. Pioneering Treatments for Rare Diseases: PTC Honors Rare Disease Day 2023

    With cutting-edge biotechnology platforms, PTC has been targeting the root causes of rare disorders for 25 years. The company’s patient-centric culture began with the Duchenne community and has expanded to include aromatic L-amino acid decarboxylase deficiency (AADC-d), Huntington’s disease, spinal muscular atrophy, and many more. Since Rare Disease Day was created in 2008, PTC employees have…

  2. Introducing Insightful Moments™, a Rare Disease Patient Education Series

    En español haz clic aquí Living with a rare disease or caring for someone with a rare disease comes with many challenges. In addition to processing new and complicated information and making countless decisions, it can be tough to know where to turn to for help, or where to even begin. To help support patients…

  3. Gene Therapy: A New Paradigm in Rare Diseases

    This article has been extracted from the special “Grand Angle dossier” on rare diseases, produced by CommEdition, published in Le Monde on 27 February 2021. It has been translated to English (below). You can also download the original French publication from this webpage. The emergence of a new generation of treatments in biotechnology has aroused…

  4. Our CEO Has Big Plans

    PTC founder and Chief Executive Officer, Stuart Peltz, Ph.D., was recently the subject of a MassBio profile. When asked about the future of PTC, he had this to say: PTC has been growing rapidly over the last two years and we will continue to grow and expand in the upcoming year. We plan to launch…