PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy

Clinical care and research in cystic fibrosis (CF) have been heavily influenced by the emergence of highly effective modulator drug therapies (HEMT) that target the CF transmembrane conductance regulator protein (CFTR). These CFTR modulators increase CFTR function by improving the processing, trafficking, and gating of aberrant proteins in the cell. Results show that CFTR modulators significantly, and at times dramatically, improve health outcomes and quality of life [1,2]. Herein we highlight a breadth of observational research studies now being conducted to understand the impact of HEMT regimens across a spectrum of disease manifestations.

Source: Journal of Cystic Fibrosis - February 19, 2021 Category: Respiratory Medicine Authors: Dave P. Nichols, Scott H. Donaldson, Carla A. Frederick, Steven D. Freedman, Daniel Gelfond, Lucas R. Hoffman, Andrea Kelly, Michael R. Narkewicz, Jessica E. Pittman, Felix Ratjen, Scott D. Sagel, Margaret Rosenfeld, Sarah Jane Schwarzenberg, Pradeep K. S Tags: Review Source Type: research