News, talk and articles
The latest news from PTC
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What You Need to Know About Ferroptosis & Friedreich’s Ataxia (FA)
An Informative Q&A with Jonathan Cherry, Global Project Leader Our research efforts at PTC are focused on two scientific platforms – splicing and inflammation and 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… -
What You Need to Know About Ferroptosis
Our research efforts at PTC are focused on two scientific platforms – splicing and inflammation and 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… -
A Whole Team in One Molecule: PTC’s Splicing Platform
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… -
An Important Job and Rewarding Career
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… -
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 joins Screen4Care to shorten the diagnosis journey for people living with rare diseases
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…