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PTC in 2021: A Year of Expansion

December 22, 2021

  1. PTC in 2021: A Year of Expansion

    For PTC, 2021 was about expansion – not just literally, as we grew our global reach and added team members, but what we can do for patients, too, through our awareness efforts and continued dedication to scientific innovation. Expansion by definition is the act of becoming larger or more extensive. And in fact, we had so much good news in 2021, it was hard for us to pick the highlights to include…

  2. Ever Better: Striving to Improve Clinical Trials for Patients

    Recently, Clinical Leader published a guest column by PTC Chief Development Officer Matthew B. Klein, MD, MS, FACS. Dr. Klein brings many years of experience working directly with patients undergoing hospital treatments and clinical studies to his work at PTC. His honed perspective has led him to identify three ways our life sciences industry can…

  3. Understanding Gene Therapy

    At PTC, we study human genes with a mission to treat rare genetic disorders. Our scientists are excited about the possibilities of gene therapy when it comes to treating rare diseases. However, we understand that when people first think about gene therapy, they may have questions. What is it? How does it work? What problems…

  4. News About PTC Gene Therapy Published by the European Molecular Biology Organization (EMBO)

    PTC is excited to announce the publication of “Gene therapy in the putamen for curing AADC deficiency and Parkinson’s disease” in the EMBO Molecular Medicine journal online with open access. Read the Article Featuring collaboration from medical experts Paul Wuh-Liang Hwu, Karl Kiening, lrina Anselm, David R. Compton, Takeshi Nakajima, Thomas Opladen, Phillip L. Pearl,…

  5. Targeting the Regulation of Protein Production for New Medicines

    The complexity of protein production associated with protein disorders, especially from faulty genes, is formidable. But that hasn’t stopped PTC from innovating in the space of RNA targeting for novel medicines. In 2003, PTC discovered the first novel small molecule capable of suppressing premature termination of translation by allowing the ribosome to selectively read through…

  6. Enrolling Your Child in a Clinical Trial – Get the Basics

    Key Takeaways • PTC clinical trials are regulated and reviewed by the FDA in the United States, and by local regulatory authorities, ethics committees, and institutional review boards in respective markets • Most patients in PTC trials see an independent doctor for evaluation before enrolling • PTC trial staff and researchers monitor individual patient data…

  7. PTC Hosts Interactive Panel Discussion on Gene Therapy Policy in Europe

    PTC Therapeutics teamed up with the Alliance for Regenerative Medicine (ARM) to host an interactive panel discussion on cell and gene therapies for rare diseases. This event explored how policy leaders can work together to advance access to cell and gene therapy in Europe. Watch the video! The growing number of advanced therapy medicinal products…

  8. 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…

  9. The Future of Rare Disease Therapies

    PTC Chief Development Officer, Matthew Klein, MD, MS, FACS recently discussed gene therapies, the impact of COVID-19, and regulatory processes with International Clinical Trials (ICT). Download the article, or read the highlights here. — ICT: How do you see the landscape of gene therapy trials for rare diseases looking in the future? Dr. Matthew Klein:…

  10. PTC to Celebrate Rare Disease Day 2021

    Every year, the rare disease community comes together to mark their collective journey with a day for recognition, sharing and reflection on the last day of February, Rare Disease Day. PTC is honored to observe this awareness day in unity with our rare disease community patients, caregivers and advocacy partners. Our SVP, Corporate & Patient…