The latest news from PTC

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…

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…

At its core, PTC’s Patient Engagement team is deeply committed to understanding the patient experience. The team knows firsthand the importance of making direct connections and building relationships with people in the rare disease community. Working closely with patients, caregivers and patient advocacy groups, their goal is to offer support, connection, and resources that address…

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…

Last week, we presented at the 40th Annual J.P. Morgan Healthcare Conference, an annual healthcare investment symposium connecting healthcare industry leaders, innovators, and the broader healthcare industry. Even though this year’s meeting was (once again) virtual, the excitement for everything that’s to come in 2022 was palpable – especially for us at PTC, as 2022…

As the expression goes, “the journey is more important than the destination.” For Global Project Leader Neil Smith, PharmD, MBA, both are equally important, as improving the lives of other people is the ultimate destination. Smith has kept this end goal in mind throughout his journey working on PTC923 – an oral therapy in development…

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…

In 2021, PTC is a silver sponsor for the Global PKU Patient Conference in September. We had a chance to catch up with Monique Cooper – the conference chairwoman and president of the Metabolic Dietary Disorders Association in Victoria, Australia –  to learn a little more about phenylketonuria (PKU) and how this patient-facing conference plans…

Heidi Maxfield is mother to a son with phenylketonuria (PKU) and sits on the Board of Directors at Intermountain PKU & Allied Disorders (IPAD). The following article was contributed to the PTC Newsroom to help spread awareness about PKU. Reach Heidi on Facebook or Instagram @maxfam. Do you have a rare disease story to share?…

PTC has successfully completed acquisition of Censa Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic rare diseases, including phenylketonuria (PKU) and other rare diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth. “This acquisition adds a well-established late-stage PKU clinical…