The latest news from PTC

On May 20, we recognize International Clinical Trials Day: a time to honor everyone who makes clinical research possible, especially the people living with rare diseases who choose to participate in clinical studies. For the rare disease community, clinical trials are more than a scientific milestone. They can mean extra appointments, travel, tests, and uncertainty –…

An Informative Q&A with Jonathan Cherry, Global Project Leader Our research efforts at PTC are focused on two scientific platforms – splicing and inflammation/ferroptosis – where PTC has unique expertise to discover and advance innovative therapies to the clinic. We recently sat down with Jonathan Cherry, Global Project Leader, to learn more about ferroptosis and…

Our research efforts at PTC are focused on two scientific platforms – splicing and inflammation/ferroptosis – where PTC has unique expertise to discover and advance innovative therapies to the clinic. We recently sat down with Jeff Trimmer, Senior VP, Research Site Head, to learn more about ferroptosis, how it relates to inflammation and the team…

There are many things that make PTC Therapeutics truly unique, and the splicing platform is one of them. Here, “splicing” refers to PTC’s scientific platform that takes aim at modulating pre-messenger RNA (pre-mRNA) splicing to control protein production. This scientific platform together with the technology that powers it, enables the identification of small molecules, which…

It’s Calum Sinclair’s job to help ensure patient safety. As the Vice President, Global Pharmacovigilance (PV) at PTC, Calum leads a global team who collect, assess, and monitor the use and safety of PTC’s medicines, from clinical to commercial setting.   It’s a highly important role that Calum candidly enjoys and has throughout his extensive career…

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…

For too many people living with rare diseases, getting a correct diagnosis is a lengthy process that negatively impacts patients and their family. Through an innovative research collaboration called Screen4Care, PTC will be working alongside 36 international partners from academia, industry, and patient advocacy organizations to find solutions to shorten the path to diagnosis and…