
In the past decade, the medical management of people with cystic fibrosis (pwCF) has changed with the development of small molecules that partially restore the function of the defective CF transmembrane conductance regulator (CFTR) protein called CFTR modulators. HEMTs are responsible for rapid respiratory manifestations, including improvement in symptoms and lung function and reduction in the rate of pulmonary exacerbations. Recent clinical trials and real-world studies suggest that the benefits of HEMT could even prove greater when used earlier in life. This article shortly reviews the past ten years of development and use of CFTR modulators. Effects of HEMT on extrapulmonary manifestations and CF demographics are also discussed.
Like
Save
Share