News, talk and articles
The latest news from PTC
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In it Together: Rare Disease Day 2024
Rare Disease Day, which takes place every year on the last day of February, is a global movement aimed at raising awareness of the 7,000+ rare diseases and more than 300 million people worldwide who live with a rare disease. With our deep experience in the rare disease space for more than 25 years and… -
AADCAware Registry: An Example of the Value that Disease Registries Bring to AADC Deficiency
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… -
PTC Italy Wins Two Prestigious Awards for Innovation and Leadership
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… -
Kelly Heger: A Pioneering Advocate
Kelly Heger became a nurse because of her daughter, Jillian. When Jillian was a baby, Kelly and her husband, Bruce, knew something was wrong. She had a very low temperature, and soon she began experiencing what is called an oculogyric crisis; Jillian’s eyes would roll upward and her neck would forcefully turn to one side… -
Committed to Doing our Part: 2022 Corporate Sustainability Report Highlights
Last week, we were thrilled to publish the PTC 2022 Corporate Sustainability Report. “Committed to doing our part” is a comprehensive look at our science, support for rare disease patient communities and access to care, our strengths-based learning culture, global equality, diversity and inclusion, efforts to help the environment, and much more. As a growing… -
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… -
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… -
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… -
National Family Caregivers Month: Thank You, Caregivers
National Family Caregivers Month (NFCM), which takes place each November, is a time to recognize and express gratitude for the more than 50 million Americans who provide care and medical assistance to their loved ones, including parents, children and siblings. We’d like to take this time to thank caregivers in the U.S. for their selfless… -
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…