ResearchCon 2020 | Wait – CFTR isn’t Just in the Lungs?

This session focused on what cystic fibrosis transmembrane conductance regulator (CFTR) does outside of the lungs, starting with how CFTR causes non-pulmonary symptoms, followed by personal and research insights into how “switching on” CFTR changes those symptoms.

Speakers also discuss the PROMISE and BEGIN studies, which will help us understand more about the effects of modulators outside the lungs. What we know so far from ivacaftor (Kalydeco) will help us understand what may happen in the era of Trikafta, so speakers also discuss their personal observations about changes in the body on modulators and how these observations help drive research.

In addition to people who are eligible for modulators, people who are not eligible for modulators will also benefit from this session, since the more we learn about what CFTR does outside the lungs, the better we will be able to design the path to a cure.

Originally recorded at ResearchCon on April 16, 2020.