Calling all 5Ts: Update on the Kalydeco/Residual CFTR study in Denver

Another significant step forward for the efforts of the Smackdown: After months of efforts lobbying for the addition of the IVS8-5T genotype to the Ivacaftor / Residual CFTR study in Denver, we were just notified that Vertex, and the study center, has agreed to recruit candidates with this genotype. 

If you carry a major CF mutation and the 5T variant (TG11, TG12, or TG13) and otherwise qualify for this study, please contact us, and we'll help connect you.

Disclaimer: While the fact that a daughter of a Smackdown founder carries the 5T obviously puts this variant on our radar, it is also carried by nearly 10% of the population, so we think it's worth paying some attention to. We will write a blog post later on the significant of the 5T, but suffice to say, if a drug like Kalydeco can potentiate residual 5T CFTR, there could be broad implications across a broad spectrum of disease.