News, talk and articles
The latest news from PTC
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25 Remarkable Stories: 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… -
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… -
Honoring AADC Deficiency Awareness Day 2022
For the third year, PTC will commemorate AADC Deficiency Awareness Day on Sunday, Oct. 23. This groundbreaking event was established in 2020 and spearheaded by AADC deficiency caregiver and founder of the AADC Family Network, Kelly Heger. The official designation of AADC Deficiency Awareness Day has enabled parents and caregivers, patient advocacy groups, legislators and… -
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…