The latest news from PTC

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…

Ron Bartek feels that maybe he and his wife Raychel were meant to establish an organization with a mission to treat and cure Friedreich Ataxia (FA). Ron first learned about FA when his son was diagnosed with the rare disease in 1997, after he was showing some signs of physical awkwardness. When Ron and Raychel…

When Kyle Bryant first started riding his recumbent trike, he had no idea he’d end up rallying a community to raise $11 million dollars for Friedreich ataxia (FA) research – he just wanted to make it down the block. Ultimately what drove Kyle, Director of rideATAXIA at FARA and Cohost of the Two Disabled Dudes…

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…

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…

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…

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…

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…